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Archive for the ‘Bone Marrow Stem Cells’ Category

Bone Marrow Stem Cell Transplant HSCT : National …

In January 2019, an international team of researchers led by Richard K. Burt, MD (Northwestern University, Chicago, IL) published results of the first randomized, control trial of bone marrow stem cell transplant (HSCT) in people with aggressive relapsing-remitting MS. They enrolled 110 people whose MS was not controlled by available disease-modifying therapies. Half received immunosuppressant therapy followed by hematopoietic (blood cell-producing) stem transplant. The other half were switched to a different disease-modifying therapy. Significantly fewer people experienced MS progression in the group that underwent HSCT, compared with the group who were switched to a different MS disease-modifying therapy. There were no deaths or life-threatening adverse events in either group. The investigators consider this study to be preliminary and recommend that further research is needed to confirm these findings and to determine longer-term outcomes and safety. Read the summary or read the abstract in JAMA.

In December 2018, Drs. John Moore, David Ma (St. Vincents Hospital, Darlinghurst, NSW, Australia) and colleagues reported results of a small clinical trial of HSCT conducted at a single medical center in Australia. This trial enrolled 35 people with relapsing-remitting MS or secondary progressive MS whose disease had not responded well to disease-modifying medications. There was no control group or blinding; all participants underwent the HSCT procedure. The team reported on results after following participants from 12 to 66 months after transplantation. After 12 months, 82% remained free of relapses, MRI-detected new or enlarging lesions, and progression (called Event-Free Survival or EFS). At two years after transplant, 65% of the group had EFS, and at three years 60%. EFS was better in those who had relapsing MS. Of 8 who experienced MS progression after transplantation, 2 had relapsing-remitting MS and 6 had secondary progressive MS. Twelve of thirteen whose disability scores improved after transplantation had relapsing-remitting MS.At this center, which has a long experience with bone marrow transplants, there were no transplant-related deaths. Many experienced complications expected from the chemotherapy cocktail (called BEAMS) used to deplete their bone marrow cells in preparation for the transplant. Read a summary or read the abstract in the JNNP.

In April 2017, researchers in Italy combined and analyzed results from 15 previously published studies of HSCT (Hematopoietic Stem Cell Transplantation) involving 764 people with various forms of MS. They found that overall, the procedure showed a significant benefit against disease activity and progression. Two years after transplantation, about 83% of all participants had not progressed; overall, studies involving more people with relapsing-remitting MS had lower progression rates. The pooled results showed an overall transplant-related mortality rate of 2.1%.There were fewer deaths in later studies as researchers gained more experience with the procedure. Read a summary of more details here or the abstract in Neurology

In February 2017, results of an international study were published. The study evaluated long-term outcomes from HSCT in 261 people with different forms of MS. The transplants took place between 1995 and 2006, with a follow-up period of up to 16 years. Several different transplant protocols were followed. After 5 years, 46% still had not experienced any progression or worsening of symptoms, including 73% of those with relapsing MS and 33% of those with secondary progressive MS. Eight deaths (2.8%) occurred within 100 days of the transplant. Most of these occurred during the early development of the procedure; improvements in patient selection and transplant techniques have significantly reduced the mortality. Those with the best outcomes tended to be younger, had relapsing MS, lower accumulation of disability and had used fewer MS therapies prior to the transplant procedure. Additional research is needed to better understand who might benefit from this procedure and how it compares to the benefits of powerful immune-modulating therapies now available. A phase 3 trial of HSCT is now in planning stages. The Society is engaged with the team planning the trial and is encouraging quick action to design and launch the trial.Read a summary of the results or the paper in JAMA Neurology

In February 2017, results were published from a multi-center, 5-year trial called theHALT MS Study. It tested HSCT in 24 people with MS and active relapsing-remitting disease that was not controlled by disease-modifying medications. Results suggest that after five years, 69.2% of participants experienced no new disease activity after the procedure and did not need disease-modifying therapies to control their disease. All participants experienced severe and/or life threatening adverse events. Most of these occurred within the first 30 days after transplant and were related to low white blood cell counts and infections. This trial, which was funded by the National Institutes of Health, is an important addition to research needed to determine whether this approach to stem cell transplantation is safe and effective in people with MS. A larger, phase 3 trial is in planning stages.Read a summary of the results or the paper in Neurology

In June 2016 researchers in Canada published results of a long-term HSCT trial involving 24 people with aggressive relapsing-remitting MS whose disease was not controlled with available therapies. Three years after the procedure, 70% remained free of disease activity, with no relapses, no new MRI-detected inflammatory brain lesions, and no signs of progression. None of the surviving participants experienced clinical relapses or required MS disease-modifying therapies to control their disease, and 40% experienced reductions in disability. One participant died and another required intensive hospital care for liver complications. All participants developed fevers, which were frequently associated with infections, and other toxicities.Read more about this study

In October 2015, researchers at the University of Genoa and other institutions in Italy reported on a small trial of HSCT in seven people with very active relapsing-remitting MS that was not controlled with MS disease-modifying therapy. They underwent a low-intensity lympho-ablative regimen in which the immune system was suppressed but not completely depleted before the stem cell transplant as an approach to reducing toxicity. The investigators did MRI scans (for 3 years) and clinical evaluations (for 5 years). They found dramatic reductions of MRI-detected inflammation after the procedure, but did not achieve complete absence of inflammation. After 5 years, two participants remained stable, one significantly improved, and four had mild disease progression. One experienced a relapse after treatment. No severe side effects occurred. The authors conclude that the low-intensity regimen they used was not sufficient to treat aggressive MS.Read an abstract from the paper(Multiple Sclerosis 2015 Oct;21(11):1423-30) In January 2015, doctors at Northwestern University published their10-year experience of treating people with HSCT. The report included 123 people with relapsing-remitting MS and 28 with secondary-progressive MS. Their method is nonmyeloblative HSCT, in which the immune system is suppressed but not completely depleted before the stem cell transplant. Individuals were followed from 6 months to 5 years, or an average of 2.5 years. The EDSS disability scores improved, compared to pretreatment, by one point or more in 64% of those followed out to year 4. Relapses and MRI-detected disease activity were also reduced. In evaluating which type of individuals benefited from the therapy, the doctors suggested that people with relapsing-remitting MS who had had MS for ten years or less showed improvements in their disability scores, whereas those with secondary-progressive MS or disease duration greater than ten years did not show improvements on their disability scores. They reported no treatment-related deaths or serious infections. ITP (immune-mediated thrombocytopenia), a potentially serious bleeding disorder, developed in 7 people, and thyroid disorders developed in 7 people.Read a summary of their resultsor thepaper in JAMA (Published onlineJanuary 20, 2015).

Ongoing Research in HSCTAdditional research is focusing on figuring out who might benefit from this procedure and how to reduce its risks. HSCTis being investigated in Canada, the United States, Europe and elsewhere. For example:

Dr. Richard Burt of Northwestern University in Chicago has recently begun a new phase 3 clinical trial at Northwestern to try to determine the optimal protocol for safety and benefit. Read more about this trial on clinicaltrials.gov A clinical trial is getting underway at medical centers in Denmark, Netherlands, Norway and Sweden. The trial is testing treatment with HSCT compared with alemtuzumab in people with active relapsing-remitting MS. Read more about this trial on clinicaltrials.gov

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Bone Marrow Stem Cell Transplant HSCT : National ...

Bone Marrow Stem Cells Stall Out in Chronic Lymphocytic …

Snow and ice cause cars to stall out on the road to their destination. In patients with CLL, its their stem cells that stall out and researchers want to know why.

For patients who have chronic lymphocytic leukemia, fighting off a serious infection can be difficult and often is just not possible. And a team of Mayo researchers is starting to find out why in a paper published recently in the journal Leukemia.

What is Chronic Lymphocytic Leukemia?

This disease is cancer of an immune cell called a B lymphocyte. These cells form in bone marrow and migrate out to patrol in the blood stream and lymphoid organs. But in chronic lymphocytic leukemia, the immune system is depleted, a state called immunodeficiency. Because of that, people with this type of leukemia are prone to serious infections and the diseases those may cause. They are also prone to developing other types of cancer.

And its those resulting problems that may ultimately contribute to death explains Kay Medina, Ph.D., a Mayo Clinic immunologist. Dr. Medina specializes in how immune cells develop from bone marrow stem cells.

In our bone marrow, stem cells convert to red blood cells, platelets or a variety of immune cells. Those are then sent into the blood stream where they do their job. Red blood cells replace cells that are worn out.

White blood cells patrol the byways of our circulation, chasing down everything from cellular debris to bacteria to virus particles.But not in patients with chronic lymphocytic leukemia.

Joining the Team

Research on chronic lymphocytic leukemia is going on in several labs at Mayo Clinic. Dr. Medina got involved after speaking with colleagues Wei Ding, M.B.B.S, Ph.D., and Neil Kay, M.D., both chronic lymphocytic leukemia physician researchers.

Mayo has a strong tradition of encouraging physician/basic research collaborations to advance knowledge of disease mechanisms, development, and assessment of new treatment approaches, says Dr. Medina.

The basic research helps us understand the cause of the disease, in this case the leukemia cell, but it also helps to understand what the disease does to other parts of the body, such as the lymph nodes, spleen, blood and bone marrow, she says.

Bone marrow is the organ that replenishes all cells in the immune system but has not been evaluated for functional proficiency in CLL patients, explains Dr. Medina.

Checking out the Cells and their Environment

Kay Medina, Ph.D.

Dr. Medinas team, with funding from Mayo Clinics Center for Biomedical Discovery, decided to look at bone marrow stem cells and their ability to generate all blood cell types. Some of the immune deficiency may be the result of treatment, but untreated patients have the same problem. The chronic nature of the disease itself may also dampen immune activity. But Dr. Medina explains that the leukemia cells may promote an environment that suppresses immune function.

Our research seeks to add to the discussion by identifying additional ways patients with CLL are unable to fight off tumors and other diseases, says Dr. Medina.

In a paper published late last year, Dr. Medina and her team, including first author Bryce Manso who is a student in the Mayo Clinic Graduate School of Biomedical Sciences, examined bone marrow and blood samples from chronic lymphocytic leukemia patients and healthy controls to determine the frequency of bone marrow stem cells in each sample and how well they did their job.

Bryce Manso, presenting a poster to a conference attendee.

The authors reported that, in general, samples from patients with chronic lymphocytic leukemia have fewer stem cells in their bone marrow, and those stem cells that remain work less well than stem cells from controls.

Stalled-Out Bone Marrow Stem Cells

As to why this happens, the authors found that it was linked to loosening controls for the on/off switches which regulate this process, proteins called transcription factors. These proteins regulate key functions in the cell, and are out of whack in samples from chronic lymphocytic leukemia patients. They may prevent bone marrow stem cells from pursuing a pathway for development; stalling-out their ability to differentiate, resulting in decreased production of important blood cells that provide the first line of defense against infectious agents.

But, Dr. Medina cautions, there is more to this story.

This is an emerging area of research in that its both a unique explanation for the clinical problem of immune deficiency and it has been minimally studied, says Dr. Medina. Future studies are planned to look at specific transcription factors that control stem cell differentiation as well as how the presence of leukemic cells in the bone marrow alter blood cell development. They will then relate this information to clinically relevant complications reported in chronic lymphocytic leukemia patients, she says.

Basic Research to Improve Patient Care

Dr. Medina, her team, and their clinical colleagues hope that by understanding how bone marrow function is impaired in chronic lymphocytic leukemia patients, they can develop unique strategies to boost bone marrow function or find alternate treatments that do not block or modify marrow function.

Through this work we hope to find ways to reduce infections and the incidence of second cancers in chronic lymphocytic leukemia patients. Our research has the potential to improve quality of life as well as extend the lives of these patients says Dr. Medina.

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Tags: basic science, blood cancer, cancer, Center for Biomedical Discovery, chronic lymphocytic leukemia, Findings, immunology, Kay Medina, leukemia, Mayo Clinic Cancer Center, Neil Kay, News, Progress Updates, Wei Ding

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Bone Marrow Stem Cells Stall Out in Chronic Lymphocytic ...

What is a stem cell or bone marrow transplant? | non …

You might have a stem cell or bone marrow transplant as part of your treatment for non-Hodgkin lymphoma (NHL). Find out how a transplant works and why you might have it.

A transplant allows you to have high doses of chemotherapy and other treatments. The stem cellsare collected from the bloodstream or the bone marrow.

Stem cells are very earlycells made inthe bone marrow. Bone marrow is a spongy material that fills the bones.

These stem cells develop into red blood cells, white blood cells and platelets.

Red blood cells contain haemoglobin which carries oxygen around the body. White blood cells are part of your immune system and help to fight infection. Platelets help to clot the blood to prevent bleeding.

These stem cells develop into red blood cells, white blood cells and platelets.

You have a stem cell transplant after very high doses of chemotherapy. You might have targeted drugs with the chemotherapy. You may also have radiotherapy to your whole body. This is called total body irradiation or TBI.

The radiotherapy and chemotherapy has a good chance of killing the lymphomacells. But it also kills the stem cells in your bone marrow.Soyour team either collects:

After the treatment you have the stem cells into your bloodstreamthrough a drip. The cells find their way back to your bone marrow where theystart making blood cells again and your bone marrow slowly recovers.

The main difference between a stem cell and bone marrow transplant is whether stem cells are collected from the bloodstream or bone marrow.

A stem cell transplant uses stem cells from your bloodstream, or a donors bloodstream. This is also called a peripheral blood stem cell transplant.

A bone marrow transplant uses stem cells from your bone marrow, or a donors bone marrow.

Stem cell transplants are the most common type of transplant. Bone marrow transplants are not used as much. This is because:

You might have a bone marrow transplant if collecting stem cells has been difficult in your situation.

The aim of NHL treatment is usually to put it into remission. Remission means there is no sign of lymphoma.

Your doctor might suggest a transplant if your NHL:

High dose chemotherapy and a transplant aims to cure some types of NHL. Or it might control the lymphoma for longer if a cure is not possible.

Depending on your situation, you might have a transplant using:

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What is a stem cell or bone marrow transplant? | non ...

Steps of PBSC or bone marrow donation – Be The Match

Step 1: Get ready to donate

Once you join the Be The Match Registry, you will be included in patient searches every day. If you match a patient, you will be contacted to confirm that you are willing to donate. If you agree to move forward, you will be asked to update your health information and participate in additional testing to see if you are the best match for the patient. If you are the best match, you will:

There are two methods of donation: PBSC and bone marrow. The patients doctor will choose which one is best for the patient.

The time it takes for a donor to recover varies. It depends on the person and type of donation. Most donors are able to return to work, school and other activities within 1 to 7 days after donation. Be The Match considers donor safety a top priority and will follow up with you regularly until you are able to resume normal activity.

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Steps of PBSC or bone marrow donation - Be The Match

An injectable bone marrowlike scaffold enhances T cell …

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Bone Marrow for Spine and Orthopaedic Stem Cell Treatment …

Stem cells are the next frontier in the treatment of orthopaedic and spinal disorders, and the Cary Orthopaedics team is leading the way.

Using stem cells harvested from an adult patients own bone marrow,Dr. Sameer Mathurand Dr. Nael Shanti both board-certified orthopaedic spinal surgeons have developed a minimally invasive remedy for those suffering from degenerative disc disease, back pain and spinal arthritis. Applying a similar approach, Cary OrthosDr. Douglas Martini a fellowship-trained, board-certified orthopaedic surgeon specializing in sports medicine has crafted a pain-relief solution for patients living with osteoarthritis and soft tissue injuries.

Multiple research studies have shown a significant reduction in low back and joint pain and improved function after stem cell injections. While these treatments are new, 80% to 90% of patients are already reporting improvement in their symptoms after orthopaedic stem cell treatments.

Many patients suffering from degenerative disc diseases or low back pain are often not ideal candidates for surgery, and some who have chosen to undergo surgery have had unsatisfactory results. Therefore, the typical remedy for chronic orthopaedic conditions is extensive physical therapy combined with oral anti-inflammatory medications. The result: The majority of patients still had to live with pain.

Physicians at Cary Orthopaedics are utilizing orthopaedic stem cell treatment using the patients own bone marrow, the soft, spongy tissue found in the center of bones. Bone marrow in adults contains a rich reservoir of multipotent stem cells also known as Mesenchymal Precursor Cells (MPCs) that can be extracted from the patients pelvis or hip bone. Due to their unique, regenerative composition, these cells can become various types of tissues including soft tissue, bone or cartilage, which make them an excellent resource for repairing and rebuilding damaged tissue, accelerating the healing process and improving overall function.

Thanks to advancements in technology, the removal and harvesting process has become easier and less expensive. Since this is a minimally invasive procedure, it has fewer side effects compared to traditional surgery, and it causes minimal discomfort to the patient.

Bone marrow injections are a breakthrough for patients in pain. Dr. Martini, a sports medicine physician at Cary Orthopaedics, has been active in the sports medicine community, previously serving as team physician for the Carolina Hurricanes, numerous colleges, and local high schools. After 25 years of experience in sports medicine, he realizes the need for improved treatment options for the greying athlete. He has begun incorporating bone marrow aspirate concentrate (BAC) into the treatment of both acute and chronic soft tissue and joint-related injuries. I believe this will be equally helpful to the patient who needs to exercise for overall health benefits as it would be for those who need to stay at their peak athletic performance, says Dr. Martini.

We have found based on our research and experience that stem cell therapy can be very safe and effective when used with the appropriate patient population, said Kevin G. Morrison, PA-C, a member of Dr. Martinis team. All the feedback to this point has been quite positive, both on the process of having the procedure done as well as the early response. But ultimately long-term data will need to be compiled and critically examined.

Much of the previous research into stem cells has centered around placental stem cells, which can also adapt into other types of tissues. However, these have not performed well when put to the test for orthopaedic treatment. Bone marrow aspirate concentrate provides MPCs that can transform into osteocytes, chondrocytes and adipocytes, all of which are important in treating orthopedic conditions.

The latest research around mesenchymal stem cells, specifically bone marrow aspiration, is certainly promising. Dr. Martini will continue to collect more data and review patients responses.

Dr. Mathur has been an instrumental force in elevating the level of patient care at Cary Orthopaedic Spine Center since joining the practice in 2008. Dr. Mathur completed his medical school at the University of Pennsylvania and spinal reconstructive fellowship at the Rush University Medical Center in Chicago. He also taught at Dana Farber Cancer Institute in Boston. Over the last 10 years, in conjunction with the National Institutes of Health, he has conducted significant study of disc degeneration and analysis of the expression of genes that may damage the disc.

In the past decade, there have been several advancements in spinal surgery, but regenerative medicine is the next frontier, said Dr. Mathur. I see so many patients that have low back pain and leg pain from degenerative disc disease. For many, there is no good surgical treatment, and stem cell injections may be a viable option.

As an orthopaedic spine specialist, Dr. Mathur is not only an expert in spinal surgery but also in the diagnosis and treatment of a wide range of spinal problems. His depth of experience allows him to best determine whether a patient would benefit from physical therapy, stem cell injections or surgical intervention. When providing stem cell treatment, Dr. Mathur performs a single injection for all patients, whereas other clinics typically require multiple injections over several weeks.

There is currently extensive, ongoing research on the application of stem cell therapy and tissue regeneration, including an application for spinal cord injury and disc pathology, which is very exciting, said Dr. Shanti, who has dedicated a great deal of time researching the potential impact stem cell therapy can provide for his patients. Dr. Shanti believes stem cell therapy is the next great advancement in healthcare with an application for a wide spectrum of medical conditions.

Recently recognized as Top Orthopaedic Doctor by The Leading Physicians of the World for the outstanding patient care, Dr. Shantis in-depth experience and understanding of the spine allows him to guide his patients especially those with chronic back pain to the most appropriate path of treatment with the shared collaborative goal of pain relief. Dr. Shanti completed his spine surgery fellowship training at the prestigious New England Baptist Hospital, Tufts University program with an emphasis on minimally invasive spine surgery, and he has authored and presented multiple papers and textbooks on the advancement of minimally invasive spine surgery.

Orthopaedic stem cell treatment is an excellent solution for patients with degenerative disc disease and also those suffering from arthritis of the spine, bulging disc, low back pain, facet joint pain or disc with annular tears.

The stem cell injection is a same-day procedure that generally takes one hour to perform. The actual extraction of bone marrow takes up to 10 minutes. The bone marrow extraction site typically the back of the patients hip or pelvis bone is numbed using a mixture of local anesthetics. A suctioned syringe is attached to a long needle that reaches the posterior aspect of the hip. The patient may experience a minimal amount of discomfort during the extraction.

The sample is collected, transferred through a filter, and then placed into a centrifuge for spinning. The speed separates the stem cells and platelets from the bone marrow. This concentration of stem cells is then reintroduced into the degenerative or painful area under image guidance with fluoroscopy to confirm accurate placement.

The harvesting site will be numb for 1 to 2 hours after the procedure, so the patient will need to have transportation home. It is permissible to fly after the treatment, but this may cause increased pain or discomfort.

Stem cell therapy relies on the bodys own regenerative process to heal, which takes time. Patients have seen the benefits in two to three months after treatment; however, many have noticed improvements in symptoms sooner.

The recommended age range for the treatment is 20 to 70 years old. As the body ages, the quality and quantity of stem cells slowly decline. After age 70, patients may experience a sharper decline in stem cells, resulting in less beneficial outcomes.

If you think you might be a candidate for orthopaedic stem cell therapy treatment, contact Cary Orthopaedics to schedule a consultation.

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Become a Donor | The Bone Marrow Foundation

Jack, diagnosed with Acute Myelogenous Leukemia (AML), and his donor Kristy

To become a donor it just takes a small vial of blood or swab of cheek cells to be typed as a bone marrow/stem cell donor. There are many patients who are desperately waiting to find a donor match. You may be able to save someones life. There are donor registry sites throughout the country.

You must be between the ages of 18 and 60 and in general good health. You should be committed to helping any patient. A simple blood test or cheek cell swab that is given through an authorized National Marrow Donor Program Donor Center or Recruitment Group is needed to obtain your HLA tissue type so it can be entered into the National Registry. You will have to complete a short health questionnaire and sign a form stating that you understand what it means to be listed in the Registry.

The cost for HLA tissue typing ranges from $45 to $96 depending on the Donor Center, the level of testing performed, and the laboratory that analyzes the test results. There may be funding available to offset this cost through the Donor Center. After the initial testing, all medical expenses are covered by the recipient or the recipients insurance. Please contact your local Donor Center for further information.

To find out more information and to become a donor:

Delete Blood Cancer | DKMS1-866-340-3567www.deletebloodcancer.org

The National Marrow Donor Program/Be The Match1-800-654-1247www.marrow.org

The American Bone Marrow Donor Registry1-800-745-2452www.abmdr.org

The Gift of Life1-800-9MARROWwww.giftoflife.org

The Icla da Silva Foundation, Inc.Helping Children and Adults with Leukemia(866) FDN-ICLAwww.icla.org

Every 15 minutes, someone in the United States is diagnosed with a medical condition (over 35,000 people a year) such as leukemia, anemias, myelodysplastic disorders and other life-threatening diseases that require treatment with bone marrow/stem cell transplants. Nearly 70 percent of these patients must rely on an unrelated donor to offer them this precious gift of life. Unfortunately, many patients who are in need of a bone marrow/stem cell transplant cannot find a suitable donor no relatives that match and no match among volunteer donors.

Fortunately, there is an alternative that has been researched and is now proving to be a good option for many of these patientsstem cells from a newborns placental and umbilical cord blood. A newborns umbilical cord and placenta contains stem cells that are the building blocks for mature blood and immune system cells. Umbilical cord blood is collected at the time of birth under controlled conditions, shipped to a blood bank where it is tested, typed and stored.

Two studies published in The New England Journal of Medicine, Volume 351:2276-285 and an editorial by Miguel A. Sanz, M.D., Ph.D. in the same issue, concluded that cord blood should be considered as an acceptable source of stem cells in the absence of a matched bone marrow donor. For many gravely ill patients (who do not have an available donor who is a match), the immediate availability of typed cord blood units is a compelling reason for its use. And for ethnic minorities, who may have unique combinations of HLA types, the chances of finding a donor match with cord blood is greater than from the existing bone marrow donor pool.

If you have a family history of certain diseases you might choose to save your babys cord blood with a private bank. Alternatively, you can donate the cord blood to a public bank. The Bone Marrow Foundation encourages you to direct any questions you have concerning the use and storage of cord blood to your physician or other appropriate health care professional. The following are further resources for more information on public and private banking:

Public Banking National Marrow Donor Program1-800-654-1247www.marrow.org

National Cord Blood ProgramNew York Blood Center310 East 67th StreetNew York, NY 100211-866- 767-NCBP (6227)www.nationalcordbloodprogram.org

Parents Guide to Cord Blood Bankingwww.parentsguidecordblood.org

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Become a Donor | The Bone Marrow Foundation

Bone Marrow & Stem Cell Transplant | Weill Cornell Medicine

Bone Marrow & Stem Cell Transplant

The Bone Marrow and Stem Cell Transplant Program at Weill Cornell Medicine was established with the mission of providing the best care and most innovative research in a compassionate and comfortable environment.

We take a multidisciplinary approach to care for patients with cancer and blood diseases who need stem cell transplants, providing world-class clinical care in collaboration with experts in leukemia, lymphoma, myeloma and other blood disorders. Based at NewYork-Presbyterian/Weill Cornell Medical Center, one of the top ten general hospitals in the nation, the expertise of our consulting team is unsurpassed.

Our patients and families cope with life-threatening illness; as such, sensitivity and compassion are a priority for our team. We view each patient as an individual, and our approach ensures that each treatment regimen is narrowly tailored to meet the unique, changing needs of our patients and their families before, during and after transplant.

As New Yorks premier healthcare institution, Weill Cornell Medicine is at the forefront of scientific research and clinical trials, enabling us to provide a full range of diagnostic and treatment protocols, including the latest breakthroughs in medicine.

Our Team

Our team of internationally-recognized bone marrow transplant and stem cell surgery specialists is known for advanced work and published research in:

Treating patients with aggressive leukemia and myelodysplastic syndromes

Bridge protocols for patients with refractory lymphoma and leukemia

Novel strategies to mobilize stem cells and improve transplantation for patients with multiple myeloma, leukemia and lymphoma

Transplants for solid tumors, severe auto-immune disorders, and AIDS

Treatment

We pride ourselves on exceptional outcomes and offer patients the most advanced diagnostic methods and treatment therapies to improve quality of life, including:

Umbilical cord blood transplant

Outpatient transplant

Autologous stem cell transplant; uses stem cells extracted from the bone marrow or peripheral blood of the patients own blood

Allogeneic stem cell transplant; uses stem cells extracted from the bone marrow or peripheral blood of a matching donor

Hematopoietic stem cell transplant; used to treat certain cancers of the blood/bone marrow, including leukemia and myeloma

Matched unrelated donor stem cell transplantation through the National Donor Matching Program

Non-ablative "mini" transplants

Haplo-Cord Transplant, allowing us to find donors for all patients, regardless of age or ethnic background

Bendamustine, a therapy that is well-tolerated and has excellent response rates in patients with myeloma

Novel forms of transplant, offering hope and success to older patients with leukemia

Clinical Trials

Clinical trials are important to improve outcomes and offer new treatment options. At Weill Cornell Medicine, we conduct more studies in blood cancers than any of our regional peers, allowing us to provide our patients with access to many multi-phase clinical trials. As active members of the international cancer research community, our oncologists also collaborate with other research centers to offer patients the most promising treatments available.

Second Opinions

In concert with your referring physician, we are always available to offer a second opinion in the form of a consultation with one of our specialists.

Why Choose Us?

Our collaborative approach means our patients receive supportive, comprehensive care and the most cutting-edge stem cell therapy and treatments. This enables patients to receive the best possible transplant outcomes. Additionally, we offer more allogeneic stem cell transplants for older adults than any other center in New York City and the entire tri-state area.

For more information or to schedule an appointment, call us at 212-746-2119 or 212-746-2646.

Located in New York City, Weill Cornell Medical College is ranked among the nations best by U.S. News & World Report year after year.

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Bone Marrow & Stem Cell Transplant | Weill Cornell Medicine

What is BMC, Bone Marrow Stem Cell Therapy?

Bone Marrow Concentrate (BMC) Therapy, also known as Bone Marrow Aspirate Concentrate (BMAC) Therapy, is a promising cutting-edge regenerative therapy to help accelerate healing in moderate to severe osteoarthritis and tendon injuries. While similar to Platelet Rich Plasma (PRP) in its ability to harness the bodys ability to heal itself through the aid of growth factors, BMC also utilizes regenerative cells that are contained within a patients own bone marrow. The marrow contains a rich reservoir of pluripotent stem cells that can be withdrawn from the patients hip bone and used for the procedure. Unlike other cells of the body, stem cells are undifferentiated, meaning they are able to replicate themselves into various types of tissue.

In the past, the process of removing and harvesting these cells was often difficult and expensive. With recent medical advancements in both the aspiration of the bone marrow and harvesting of the regenerative cells, the procedure can be done with minimal discomfort and patients are sent home the same day. The process is relatively simple. The patient is first numbed using a mixture of local anesthetics. Under the guidance of an X-Ray machine, the physician then removes a small amount of the patients bone marrow from the hip bone which is then placed into a centrifuge to separate the regenerative cells and platelets from the rest of the blood products. The final product is a concentrate which has approximately 5-10 times the baseline levels of regenerative cells and growth factors. This point of care treatment allows for minimal manipulation of cells which are then injected to the injured area. The entire process takes approximately 2 hours and patients go home the same day.

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What is BMC, Bone Marrow Stem Cell Therapy?

How Bone Marrow and Stem Cells are Collected | BMT Infonet

Language English

If you are providing the blood stem cells for a transplant, they will either be collected from your bloodstream (peripheral blood) or from your bone marrow.

The largest concentration of blood stem cells is in your bone marrow. However, the blood stem cells can be moved or "mobilized" out of the bone marrow into the bloodstream (peripheral blood) where they can be easily collected. Most transplants these days use stem cells collected from the bloodstream.

When blood stem cells are collected from the bloodstream, the procedure is called a peripheral blood stem cell collection or harvest.

Prior to the harvest, you will receive injections of a drug such as filgrastim (Neupogen) or plerixifor (Mozobil) over a four to five day period. These drugs move stem cells out of the bone marrow into the bloodstream.

Most people tolerate these drugs well, although mild, flu-like symptoms are common. The symptoms end a few days after the injections stop.

If you are collecting stem cells for your own transplant, chemotherapy drugs may be used to help move the stem cells out of your bone marrow into the bloodstream.

Peripheral blood stem cell collections are done in an outpatient clinic.

The procedure is painless. However, you may feel lightheaded, cold or numb around the lips. Some people feel cramping in their hands which is caused by the blood thinning agent used during the procedure. These symptoms cease when the procedure ends.

The procedure used to collect bone marrow for transplant is called a bone marrow harvest. It is a surgical procedure that takes place in a hospital operating room. Typically it is done as an outpatient procedure.

The amount of bone marrow harvested depends on the size of the patient and the concentration of blood stem cells in your marrow.

Typically one to two quarts of marrow and blood are harvested. While this may sound like a lot, your body can usually replace it in four weeks.

When the anesthesia wears off, you may feel some discomfort in your hip and lower back for several days. The pain is similar to what you would feel if you took a hard fall and bruised your hip. You may find sitting for a long period of time or climbing stairs uncomfortable for a few days. The pain is usually relieved with acetaminophen (Tylenol).

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How Bone Marrow and Stem Cells are Collected | BMT Infonet

What is a Bone Marrow Transplant (Stem Cell Transplant …

A bone marrow transplant, also called a stem cell transplant, is a treatment for some types of cancer. For example, you might have one if you have leukemia, multiple myeloma, or some types of lymphoma. Doctors also treat some blood diseases with stem cell transplants.

In the past, a stem cell transplant was more commonly called a bone marrow transplant because the stem cells were collected from the bone marrow. Today, stem cells are usually collected from the blood, instead of the bone marrow. For this reason, they are now often called stem cell transplants.

A part of your bones called bone marrow makes blood cells. Marrow is the soft, spongy tissue inside bones. It contains cells called hematopoietic stem cells (pronounced he-mah-tuh-poy-ET-ick). These cells can turn into several other types of cells. They can turn into more bone marrow cells. Or they can turn into any type of blood cell.

Certain cancers and other diseases keep hematopoietic stem cells from developing normally. If they are not normal, neither are the blood cells that they make. A stem cell transplant gives you new stem cells. The new stem cells can make new, healthy blood cells.

The main types of stem cell transplants and other options are discussed below.

Autologous transplant. This is also called an AUTO transplant or high-dose chemotherapy with autologous stem cell rescue.

In an AUTO transplant, you get your own stem cells after doctors treat the cancer. First, your health care team collects stem cells from your blood and freezes them. Next, you have powerful chemotherapy, and rarely, radiation therapy. Then, your health care team thaws your frozen stem cells. They put them back in your blood through a tube placed in a vein (IV).

It takes about 24 hours for your stem cells to reach the bone marrow. Then they start to grow, multiply, and help the marrow make healthy blood cells again.

Allogeneic transplantation. This is also called an ALLO transplant.In an ALLO transplant, you get another persons stem cells. It is important to find someone whose bone marrow matches yours. This is because you have certain proteins on your white blood cells called human leukocyte antigens (HLA). The best donor has HLA proteins as much like yours as possible.

Matching proteins make a serious condition called graft-versus-host disease (GVHD) less likely. In GVHD, healthy cells from the transplant attack your cells. A brother or sister may be the best match. But another family member or volunteer may also work.

Once you find a donor, you receive chemotherapy with or without radiation therapy. Next, you get the other persons stem cells through a tube placed in a vein (IV). The cells in an ALLO transplant are not typically frozen. This way, your doctor can give you the cells as soon as possible after chemotherapy or radiation therapy.

There are 2 types of ALLO transplants. The best type for each person depends on his or her age, health, and the type of disease being treated.

Ablative, which uses high-dose chemotherapy

Reduced intensity, which uses milder doses of chemotherapy

If your health care team cannot find a matched adult donor, there are other options. Research is ongoing to determine which type of transplant will work best for different people.

Umbilical cord blood transplant. This may be an option if you cannot find a donor match. Cancer centers around the world use cord blood.

Parent-child transplant and haplotype mismatched transplant. These types of transplants are being used more often. The match is 50%, instead of near 100%. Your donor might be a parent, child, brother, or sister.

Your doctor will recommend an AUTO or ALLO transplant based mostly on the disease you have. Other factors include the health of your bone marrow and your age and general health. For example, if you have cancer or other disease in your bone marrow, you will probably have an ALLO transplant. In this situation, doctors do not recommend using your own stem cells.

Choosing a transplant is complicated. You will need help from a doctor who specializes in transplants. You might need to travel to a center that does many stem cell transplants. Your donor might also need to go. At the center, you will talk with a transplant specialist and have an examination and medical tests.

Before a transplant, you should also think about non-medical factors. These include:

Who can care for you during treatment

How long you will be away from work and family responsibilities

If your insurance pays for the transplant

Who can take you to transplant appointments

Your health care team can help you find answers to these questions.

The information below tells you the main parts of AUTO and ALLO transplants. Your health care team usually does the steps in order. But sometimes certain steps happen in advance, such as collecting stem cells. Ask your health care team what to expect before, during, and after a transplant.

Part 1: Collecting your stem cells

During this part, you get injections of a medication to raise your number of stem cells.Your doctor may collect stem cells through your veins using standard IVs or a catheter, which is placed in a large vein in the chest. This stays in place throughout your stay at the hospital. The catheter is used to give chemotherapy, other medications, and blood transfusions.

Time: Several days

Where it is done: Clinic or hospital building. You do not need to stay in the hospital overnight.

Part 2: Transplant treatment

You get high doses of chemotherapy, and rarely, radiation therapy.

Time: 5 to 10 days

Where it is done: A clinic or hospital. At many transplant centers, people need to stay in the hospital for the duration of the transplant, usually about 3 weeks. At some centers, a person receives treatment in the clinic and can come in every day.

Part 3: Getting your stem cells back

This part is called the stem cell infusion. Your health care team puts your stem cells back in your blood through the transplant catheter.

Time: Each infusion usually takes less than 30 minutes. You may receive more than 1 infusion.

Where it is done: A clinic or hospital.

Part 4: Recovery

You take antibiotics and other drugs. You get blood transfusions through your transplant catheter, if needed. This is also when your health care team helps with any transplant side effects.

Time: Approximately 2 weeks

Where it is done: A clinic or hospital. You might be staying in the hospital.

Part 1: Collecting stem cells from your donor

During this part, the health care team gives your donor injections of a medication to increase white cells in the blood, if the cells are collected from blood. Some donors will donate bone marrow in the operating room during a procedure which takes several hours.

Time: Varies based on how the stem cells are collected

Where it is done: A clinic or hospital

Part 2: Transplant treatment

You get chemotherapy with or without radiation therapy.

Time: 5 to 7 days

Where it is done: Many ALLO transplants are done in the hospital.

Part 3: Getting the donor cells

This part is called the stem cell infusion. Your health care team puts the donors stem cells in your blood through the transplant catheter. It takes less than 1 hour. The transplant catheter stays in until after treatment.

Time: 1 day

Where it is done: A clinic or hospital

Part 4: Recovery

During the recovery, you receive antibiotics and other drugs. This includes medications to prevent graft-versus-host disease. If needed, you get blood transfusions through your catheter. This is also when your health care team takes care of any side effects from the transplant.

After the transplant, people visit the clinic frequently at first and less often over time.

Time: It varies.For an ablative transplant, people are usually in the hospital for about 4 weeks in total.For a reduced intensity transplant, people are in the hospital or visit the clinic daily for about a week.

The words successful transplant might mean different things to you, your family, and your health care team. Below are 2 ways to measure transplant success: Your blood counts are back to safe levels. A blood count is the number of red cells, white cells, and platelets in your blood. A transplant makes these numbers very low for 1 to 2 weeks. This causes risks of:

Infection from low numbers of white cells, which fight infections

Bleeding from low numbers of platelets, which stop bleeding

Tiredness from low numbers of red cells, which carry oxygen

Doctors lower these risks by giving blood and platelet transfusions after a transplant. You also take antibiotics to help prevent infections. When the new stem cells multiply, they make more blood cells. Then your blood counts improve. This is one way to know if a transplant is a success.

It controls your cancer. Doctors do stem cell transplants with the goal of curing disease. A cure may be possible for some cancers, such as some types of leukemia and lymphoma. For other people, remission is the best result. Remission is having no signs or symptoms of cancer. After a transplant, you need to see your doctor and have tests to watch for any signs of cancer or complications from the transplant.

Talking often with your health care team is important. It gives you information to make decisions about your treatment and care. The following questions may help you learn more about stem cell transplant:

Which type of stem cell transplant would you recommend? Why?

If I will have an ALLO transplant, how will we find a donor? What is the chance of a good match?

What type of treatment will I have before the transplant? Will radiation therapy be used?

How long will my treatment take? How long will I stay in the hospital?

How will a transplant affect my life? Can I work? Can I exercise and do regular activities?

How will we know if the transplant works?

What if the transplant does not work? What if the cancer comes back?

What are the short-term side effects that may happen during treatment or shortly after?

What are the long-term side effects that may happen years later?

What tests will I need later? How often will I need them?

If I am worried about managing the costs of treatment, who can help me with these concerns?

Side Effects of a Bone Marrow Transplant (Stem Cell Transplant)

Bone Marrow Aspiration and Biopsy

Donating Bone Marrow is Easy and Important: Here's Why

How Umbilical Cord BloodCan Save Someone's Life

Bone Marrow Transplants and Older Adults: 3 Important Questions

Be the Match: About Transplant

Be the Match: National Marrow Donor Program

Blood & Marrow Transplant Information Network (BMT InfoNet) National Bone Marrow Transplant Link (nbmtLINK)

U.S. Department of Health and Human Services: Learn About Transplant as a Treatment Option

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What is a Bone Marrow Transplant (Stem Cell Transplant ...

Bone Marrow & Stem Cell Transplant | IU Health

To prepare your body for bone marrow or stem cell transplant, youll be treated with high doses of chemotherapy with or without radiation to destroy cancerous cells. Some healthy cells may also be destroyed, which can cause unpleasant side effects. These side effects typically go away after a few weeks.

Once this preparation is complete, new stem cells will be transplanted through your veins and the cells will make their way to your bone marrow. These stem cells will mature into healthy marrow, to produces healthy blood and immune cells.

Stem cells transplants can come from your own bone marrow (autologous) or a donors marrow (allogeneic). Whether autologous or allogeneic stem cells are used depends on your condition, and the procedures have some differences.

Uses your own stem cells. Before chemotherapy, your stem cells are collected by apheresis, frozen with a preservative and stored until they are needed. Because the cells are yours, theres no risk of your body rejecting the transplanted stem cells. This method is appropriate for blood-related cancers like multiple myeloma, non-Hodgkin lymphomas and Hodgkin disease, as well as certain germ-cell cancers.

Use healthy cells from a donor, when an immunological effect is needed to fight your cancer. Your donor will usually be a sibling or a strong match from the national registry. If a matched sibling or unrelated donor cannot be found, cord blood stem cells or a mismatched relative donor may be used.

The donors stem cells are collected by apheresis or from the bone marrow in a surgical procedure. Youll need to take medicines to suppress your immune system to prevent rejection and keep the donors immune cells from attacking your normal cells. Donor-cell transplant is used to treat blood-related cancers like leukemias and some lymphomas or multiple myeloma, and bone marrow failure disorders like myelodysplastic syndrome and aplastic anemia.

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Bone Marrow & Stem Cell Transplant | IU Health

All Things Stem Cell Visual Stem Cell Glossary

Stem cells: Cells that are able to (1) self-renew (can create more stem cells indefinitely) and (2) differentiate into (become) specialized, mature cell types.

Embryonic stem cells: Stem cells that are harvested from a blastocyst. These cells are pluripotent, meaning they can differentiate into cells from all three germ layers.

Embryonic stem cells are isolated from cells in a blastocyst, a very early stage embryo. Once isolated from the blastocyst, these cells form colonies in culture (closely packed groups of cells) and can become cells of the three germ layers, which later make up the adult body.

Adult stem cells (or Somatic Stem Cell): Stem cells that are harvested from tissues in an adult body. These cells are usually multipotent, meaning they can differentiate into cells from some, but not all, of the three germ layers. They are thought to act to repair and regenerate the tissue in which they are found in, but usually they can differentiate into cells of completely different tissue types.

Adult stem cells can be found in a wide variety of tissues throughout the body; shown here are only a few examples.

The Three Germ Layers: These are three different tissue types that exist during development in the embryo and that, together, will later make up the body. These layers include the mesoderm, endoderm, and ectoderm.

The three germ layers form during the gastrula stage of development. The layers are determined by their physical position in the gastrula. This stage follows the zygote and blastocyst stages; the gastrula forms when the embryo is approximately 14-16 days old in humans.

Endoderm: One of the three germ layers. Specifically, this is the inner layer of cells in the embryo and it will develop into lungs, digestive organs, the liver, the pancreas, and other organs.

Mesoderm: One of the three germ layers. Specifically, this is the middle layer of cells in the embryo and it will develop into muscle, bone, blood, kidneys, connective tissue, and related structures.

Ectoderm: One of the three germ layers. Specifically, this is the outer layer of cells in the embryo and it will develop into skin, the nervous system, sensory organs, tooth enamel, eye lens, and other structures.

Differentiation, Differentiated: The process by which a stem cell turns into a different, mature cell. When a stem cell has become the mature cell type, it is called differentiated and has lost the ability to turn into multiple different cell types; it is also no longer undifferentiated.

Directed differentiation: To tightly control a stem cell to become a specific mature cell type. This can be done by regulating the conditions the cell is exposed to (i.e. specific media supplemented with different factors can be used).

The differentiation of stem cells can be controlled by exposing the cells to specific conditions. This regulation can cause the cells to become a specific, desired mature cell type, such as neurons in this example.

Undifferentiated: A stem cell that has not become a specific mature cell type. The stem cell holds the potential to differentiate, to become different cell types.

Potential, potency: The number of different kinds of mature cells a given stem cell can become, or differentiate into.

Totipotent: The ability to turn into all the mature cell types of the body as well as embryonic components that are required for development but do not become tissues of the adult body (i.e. the placenta).

A totipotent cell has the ability to become all the cells in the adult body; such cells could theoretically create a complete embryo, such as is shown here in the early stages.

Pluripotent: The ability to turn into all the mature cell types of the body. This is shown by differentiating these stem cells into cell types of the three different germ layers.

Embryonic stem cells are pluripotent cells isolated from an early stage embryo, called the blastocyst. These isolated cells can turn into cells representative of the three germ layers, all the mature cell types of the body.

Multipotent: The ability to turn into more than one mature cell type of the body, usually a restricted and related group of different cell types.

Mesenchymal stem cells are an example of multipotent stem cells; these stem cells can become a wide variety, but related group, of mature cell types (bone, cartilage, connective tissue, adipose tissue, and others).

Unipotent: The ability to give rise to a single mature cell type of the body.

Tissue Type: A group of cells that are similar in morphology and function, and function together as a unit.

Mesenchyme Tissue: Connective tissue from all three germ layers in the embryo. This tissue can become cells that make up connective tissue, cartilage, adipose tissue, the lymphatic system, and bone in the adult body.

Mesenchyme tissue can come from all three of the germ layers (ectoderm, mesoderm, and endoderm) in the developing embryo, shown here at the gastrula stage. The mesenchyme can become bone, cartilage, connective tissue, adipose tissue, and other components of the adult body.

Hematopoietic Stem Cells: Stem cells that can create all the blood cells (red blood cells, white blood cells, and platelets). These stem cells reside within bone marrow in adults and different organs in the fetus.

Hematopoietic stem cells can become, or differentiate into, all the different blood cell types. This process is referred to as hematopoiesis.

Bone marrow: Tissue within the hollow inside of bones that contains hematopoietic stem cells and mesenchymal stem cells.

Development: The process by which a fertilized egg (from the union of a sperm and egg) becomes an adult organism.

Zygote: The single cell that results from a sperm and egg uniting during fertilization. The zygote undergoes several rounds of cell division before it becomes an embryo (after about four days in humans).

When an egg is fertilized by a sperm, the resultant single cell is referred to as a zygote.

Blastocyst: A very early embryo (containing approximately 150 cells) that has not yet implanted into the uterus. The blastocyst is a fluid-filled sphere that contains a group of cells inside it (called the inner cell mass) and is surrounded by an outer layer of cells (the trophoblast, which forms the placenta).

The blastocyst contains three primary components: the inner cell mass, which can become the adult organism, the trophoblast, which becomes the placenta, and the blastocoele, which is a fluid-filled space. The blastocyst develops into the gastrula, a later stage embryo.

Inner Cell Mass: A small group of cells that are attached inside the blastocyst. Human embryonic stem cells are created from these cells in blastocysts that are four or five days post-fertilization. The cells from the inner cell mass have the potential to develop into an embryo, then later the fetus, and eventually the entire body of the adult organism.

Cells taken from the inner cell mass of the blastocyst (a very early stage embryo) can become embryonic stem cells.

Embryo: The developing organism from the end of the zygote stage (after about four days in humans) until it becomes a fetus (until 7 to 8 weeks after conception in humans).

Models: A biological system that is easy to study and similar enough to another, more complex system of interest so that knowledge of the model system can be used to better understand the more complex system. Such systems can include cells and whole organisms.

Model organism: An organism that is easy to study and manipulate and is similar enough to another organism of interest so that by understanding the model organism, a greater understanding of the other organism may be gained. For example, rats and mice can be used as model organisms to better understand humans.

Shown are several different model organisms frequently used in laboratory studies.

Severe Combined Immune-Deficient (SCID) mouse: A mouse lacking a functional immune system, specifically lacking or abnormal T and B lymphocytes. This is due to inbreeding or genetic engineering. They are extensively used for tissue transplants, because they lack an immune system to reject foreign substances, and for studying an immunocompromised system.

Cellular models: A cell system that can be used to understand normal, or diseased, functions that the cell has within the body. By taking cells from the body and studying them outside of the body, in culture, different conditions can be manipulated and the results studied, whereas this can be much more difficult, or impossible, to do within the body.

Stem cells obtained from different tissues of the body can be used as models to study normal, or diseased, cells in these tissues.

Cell Types:

Somatic Cell: Any cell in the body, developing or adult, other than the germline cells (the gametes, or sperm and eggs).

Gametes: The cells in the body that carry the genetic information that will be passed to the offspring. In other words, these are the germline cells: an egg (for females) or sperm (for males) cell.

Other terms:

Regenerative Medicine: A field of research that investigates how to repair or replace damaged tissues, usually by using stem cells. In this manner, stem cells may be differentiated into, or made to become, the type of cell that is damaged and then used in transplants. Also see clinical trials.

Clinical trials: A controlled test of a new drug or treatment on human subjects, normally performed after successful trials with model organisms. ClinicalTrials.gov lists many stem cell clinical trials.

Stem cells have great potential to treat a wide variety of human diseases and medical conditions.

Cell Surface Marker proteins, or simply Cell Markers: A protein on the surface of a cell that identifies the cell as a certain cell type.

Somatic Cell Nuclear Transfer (SCNT): A technique that uses an egg and a somatic cell (a non-germline cell). The nucleus, which contains the genetic material, is removed from the egg and the nucleus from the somatic cell is removed and combined with the egg. The resultant cell contains the genetic material of the nucleus donor, and is turned into a totipotent state by the egg. This cell has the potential to develop into an organism, a clone of the nucleus donor.

Dolly the sheep was cloned through somatic cell nuclear transfer (SCNT). An adult cell from the mammary gland of a Finn-Dorset ewe acted as the nuclear donor; it was fused with an enucleated egg from a Scottish Blackface ewe, which acted as the cytoplasmic (or egg) donor. An electrical pulse acted to fuse the cells and activate the oocyte after injection into the surrogate mother ewe. A successfully implanted oocyte developed into the lamb Dolly, a clone of the nuclear donor, the Finn-Dorset ewe.

Clone: A genetic, identical copy of an individual organism through asexual methods. A clone can be created through somatic cell nuclear transfer.

Other stem cell glossaries:

Image creditsImages of Endoderm, Mesoderm, Ectoderm, Bone Marrow, Neurons, Cartilage, Hand Skeleton, Connective and Adipose Tissue, Gastrula, Clinical Trials, Mouse, Rat, Drosophila, C. Elegans, Arabidopsis, Sea Urchin, Xenopus, Somatic Cell Nuclear Transfer to Create Dolly and other images were taken from the Wikimedia Commons and redistributed and altered freely as they are all in the public domain. The image of Hematopoiesis was also taken from the Wikimedia Commons and redistributed according to the GNU Free Documentation License.

2009. Teisha Rowland. All rights reserved.

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All Things Stem Cell Visual Stem Cell Glossary

Bone marrow suppression – Wikipedia

Bone marrow suppressionSynonymMyelotoxicity, myelosuppression

Bone marrow suppression also known as myelotoxicity or myelosuppression, is the decrease in production of cells responsible for providing immunity (leukocytes), carrying oxygen (erythrocytes), and/or those responsible for normal blood clotting (thrombocytes).[1] Bone marrow suppression is a serious side effect of chemotherapy and certain drugs affecting the immune system such as azathioprine.[2] The risk is especially high in cytotoxic chemotherapy for leukemia.

Nonsteroidal anti-inflammatory drugs (NSAIDs), in some rare instances, may also cause bone marrow suppression. The decrease in blood cell counts does not occur right at the start of chemotherapy because the drugs do not destroy the cells already in the bloodstream (these are not dividing rapidly). Instead, the drugs affect new blood cells that are being made by the bone marrow.[3] When myelosuppression is severe, it is called myeloablation.[4]

Many other drugs including common antibiotics may cause bone marrow suppression. Unlike chemotherapy the affects may not be due to direct destruction of stem cells but the results may be equally serious. The treatment may mirror that of chemotherapy-induced myelosuppression or may be to change to an alternate drug or to temporarily suspend treatment.

Because the bone marrow is the manufacturing center of blood cells, the suppression of bone marrow activity causes a deficiency of blood cells. This condition can rapidly lead to life-threatening infection, as the body cannot produce leukocytes in response to invading bacteria and viruses, as well as leading to anaemia due to a lack of red blood cells and spontaneous severe bleeding due to deficiency of platelets.

Parvovirus B19 inhibits erythropoiesis by lytically infecting RBC precursors in the bone marrow and is associated with a number of different diseases ranging from benign to severe. In immunocompromised patients, B19 infection may persist for months, leading to chronic anemia with B19 viremia due to chronic marrow suppression.[5]

Bone marrow suppression due to azathioprine can be treated by changing to another medication such as mycophenolate mofetil (for organ transplants) or other disease-modifying drugs in rheumatoid arthritis or Crohn's disease.

Bone marrow suppression due to anti-cancer chemotherapy is much harder to treat and often involves hospital admission, strict infection control, and aggressive use of intravenous antibiotics at the first sign of infection.[citation needed]

G-CSF is used clinically (see Neutropenia) but tests in mice suggest it may lead to bone loss.[6][7]

GM-CSF has been compared to G-CSF as a treatment of chemotherapy-induced myelosuppression/Neutropenia.[8]

In developing new chemotherapeutics, the efficacy of the drug against the disease is often balanced against the likely level of myelotoxicity the drug will cause. In-vitro colony forming cell (CFC) assays using normal human bone marrow grown in appropriate semi-solid media such as ColonyGEL have been shown to be useful in predicting the level of clinical myelotoxicity a certain compound might cause if administered to humans.[9] These predictive in-vitro assays reveal effects the administered compounds have on the bone marrow progenitor cells that produce the various mature cells in the blood and can be used to test the effects of single drugs or the effects of drugs administered in combination with others.

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Bone marrow suppression - Wikipedia

How Bone Marrow and Stem Cell Transplants Work

If you or a loved one will be having a bone marrow transplant or donating stem cells, what does it entail? What are the different types of bone marrow transplants and what is the experience like for both the donor and recipient?

A bone marrow transplant is a procedure in which when special cells (called stem cells) are removed from the bone marrow or peripheral blood, filtered and given back either to the same person or to another person.

Since we now derive most stem cells needed from the blood rather than the bone marrow, a bone marrow transplant is now more commonly referred to as stem cell transplant.

Bone marrow is found in larger bones in the body such as the pelvic bones. This bone marrow is the manufacturing site for stem cells. Stem cells are "pluripotential" meaning that the cells are the precursor cells which can evolve into the different types of blood cells, such as white blood cells, red blood cells, and platelets.

If something is wrong with the bone marrow or the production of blood cells is decreased, a person can become very ill or die. In conditions such as aplastic anemia, the bone marrow stops producing blood cells needed for the body. In diseases such as leukemia, the bone marrow produces abnormal blood cells.

The purpose of a bone marrow transplant is thus to replace cells not being produced or replace unhealthy stem cells with healthy ones. This can be used to treat or even cure the disease.

In addition to leukemias, lymphomas, and aplastic anemia, stem cell transplants are being evaluated for many disorders, ranging from solid tumors to other non-malignant disorders of the bone marrow, to multiple sclerosis.

There are two primary types of bone marrow transplants, autologous and allogeneic transplants.

The Greek prefix "auto" means "self." In an autologous transplant, the donor is the person who will also receive the transplant. This procedure, also known as a "rescue transplant" involves removing your stem cells and freezing them. You then receive high dose chemotherapy followed by infusion of the thawed out frozen stem cells. It may be used to treat leukemias, lymphomas, or multiple myeloma.

The Greek prefix "allo" means "different" or "other." In an allogeneic bone marrow transplant, the donor is another person who has a genetic tissue type similar to the person needing the transplant. Because tissue types are inherited, similar to hair color or eye color, it is more likely that you will find a suitable donor in a family member, especially a sibling. Unfortunately, this occurs only 25 to 30 percent of the time.

If a family member does not match the recipient, the National Marrow Donor Program Registry database can be searched for an unrelated individual whose tissue type is a close match. It is more likely that a donor who comes from the same racial or ethnic group as the recipient will have the same tissue traits. Learn more about finding a donor for a stem cell transplant.

Bone marrow cells can be obtained in three primary ways. These include:

The majority of stem cell transplants are done using PBSC collected by apheresis (peripheral blood stem cell transplants.) This method appears to provide better results for both the donor and recipient. There still may be situations in which a traditional bone marrow harvest is done.

Donating stem cells or bone marrow is fairly easy. In most cases, a donation is made using circulating stem cells (PBSC) collected by apheresis. First, the donor receives injections of a medication for several days that causes stem cells to move out of the bone marrow and into the blood. For the stem cell collection, the donor is connected to a machine by a needle inserted in the vein (like for donating blood). Blood is taken from the vein, filtered by the machine to collect the stem cells, then returned back to the donor through a needle in the other arm. There is almost no need for a recovery time with this procedure.

If stem cells are collected by bone marrow harvest (much less likely), the donor will go to the operating room and while asleep under anesthesia and a needle will be inserted into either the hip or the breastbone to take out some bone marrow. After awakening, there may be some pain where the needle was inserted.

A bone marrow transplant can be a very challenging procedure for the recipient.

The first step is usually receiving high doses of chemotherapy and/or radiation to eliminate whatever bone marrow is present. For example, with leukemia, it is first important to remove all of the abnormal bone marrow cells.

Once a person's original bone marrow is destroyed, the new stem cells are injected intravenously, similar to a blood transfusion. The stem cells then find their way to the bone and start to grow and produce more cells (called engraftment).

There are many potential complications. The most critical time is usually when the bone marrow is destroyed so that few blood cells remain. Destruction of the bone marrow results in greatly reduced numbers of all of the types of blood cells (pancytopenia). Without white blood cells there is a serious risk of infection, and infection precautions are used in the hospital (isolation). Low levels of red blood cells (anemia) often require blood transfusions while waiting for the new stem cells to begin growing. Low levels of platelets (thrombocytopenia) in the blood can lead to internal bleeding.

A common complication affecting 40 to 80 percent of recipients is graft versus host disease. This occurs when white blood cells (T cells) in the donated cells (graft) attack tissues in the recipient (the host), and can be life-threatening.

An alternative approach referred to as a non-myeloablative bone marrow transplant or "mini-bone marrow transplant" is somewhat different. In this procedure, lower doses of chemotherapy are given that do not completely wipe out or "ablate" the bone marrow as in a typical bone marrow transplant. This approach may be used for someone who is older or otherwise might not tolerate the traditional procedure. In this case, the transplant works differently to treat the disease as well. Instead of replacing the bone marrow, the donated marrow can attack cancerous cells left in the body in a process referred to as "graft versus malignancy."

If you'd like to become a volunteer donor, the process is straightforward and simple. Anyone between the ages of 18 and 60 and in good health can become a donor. There is a form to fill out and a blood sample to give; you can find all the information you need at the National Marrow Donor Programwebsite. You can join a donor drive in your area or go to a local Donor Center to have the blood test done.

When a person volunteers to be a donor, his or her particular blood tissue traits, as determined by a special blood test (histocompatibility antigen test,) are recorded in the Registry. This "tissue typing" is different from a person's A, B, or O blood type. The Registry record also contains contact information for the donor, should a tissue type match be made.

Bone marrow transplants can be either autologous (from yourself) or allogeneic (from another person.) Stem cells are obtained either from peripheral blood, a bone marrow harvest or from cord blood that is saved at birth.

For a donor, the process is relatively easy. For the recipient, it can be a long and difficult process, especially when high doses of chemotherapy are needed to eliminate bone marrow. Complications are common and can include infections, bleeding, and graft versus host disease among others.

That said, bone marrow transplants can treat and even cure some diseases which had previously been almost uniformly fatal. While finding a donor was more challenging in the past, the National Marrow Donor Program has expanded such that many people without a compatible family member are now able to have a bone marrow/stem cell transplant.

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How Bone Marrow and Stem Cell Transplants Work

Bone Marrow-Derived Stem Cell Therapy Milwaukee, WI …

Advanced Therapy with Advanced Results

Since 1968, the medical community has been harnessing the incredible healing, and regenerative power of bone marrow-derived stem cells. Bone Marrow Derived Stem Cell Therapy takes stem cells isolated from your bone marrow and relocates them to heal, regenerate and treat damaged areas and chronic conditions. This revolutionary technology is a result of decades of evidence-based research and advancements in the area of stem cells.

A process called hematopoiesis, which occurs inside your bones, has been working to grow and regenerate cells in your body since you were in the womb. The human body is in constant high demand for blood cells, so the hematopoiesis process stays hard at work to produce. During hematopoiesis, hematopoietic stem cells are produced with the raw potential power to develop into white blood cells, red blood cells, and platelets. Blood cells are vital to immune function and healing, so these stem cells are rich in growth factors that facilitate the repair and replacement of damaged cells. Mesenchymal stem cells are also found in bone marrow. Mesenchymal stem cells are reserved adult stem cells that help facilitate the regeneration of tissue naturally in the body. They are an integral part of wound healing, regulation of aging, and stabilizing vital organs. These mesenchymal stem cells are considered to be raw potential meaning they can differentiate into the tissue cells needed in a specific area. These mesenchymal stem cells have the potential to repair damaged cartilage, bone, tendons, muscle, skin, and connective cell tissue.

Stem cell therapy is one of the newest and most cutting-edge therapies for chronic joint pain. Using this therapy, our providers offer patients essential properties for healing and restoring joint health:

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Bone marrow transplant – Mayo Clinic

Overview

A bone marrow transplant is a procedure that infuses healthy blood stem cells into your body to replace your damaged or diseased bone marrow. A bone marrow transplant is also called a stem cell transplant.

A bone marrow transplant may be necessary if your bone marrow stops working and doesn't produce enough healthy blood cells.

Bone marrow transplants may use cells from your own body (autologous transplant) or from a donor (allogeneic transplant).

Mayo Clinic's approach

A bone marrow transplant may be used to:

Bone marrow transplants can benefit people with a variety of both cancerous (malignant) and noncancerous (benign) diseases, including:

Bone marrow is the spongy tissue inside some bones. Its job is to produce blood cells. If your bone marrow isn't functioning properly because of cancer or another disease, you may receive a stem cell transplant.

To prepare for a stem cell transplant, you receive chemotherapy to kill the diseased cells and malfunctioning bone marrow. Then, transplanted blood stem cells are put into your bloodstream. The transplanted stem cells find their way to your marrow, where ideally they begin producing new, healthy blood cells.

A bone marrow transplant poses many risks of complications, some potentially fatal.

The risk can depend on many factors, including the type of disease or condition, the type of transplant, and the age and health of the person receiving the transplant.

Although some people experience minimal problems with a bone marrow transplant, others may develop complications that may require treatment or hospitalization. Some complications could even be life-threatening.

Complications that can arise with a bone marrow transplant include:

Your doctor can explain your risk of complications from a bone marrow transplant. Together you can weigh the risks and benefits to decide whether a bone marrow transplant is right for you.

If you receive a transplant that uses stem cells from a donor (allogeneic transplant), you may be at risk of developing graft-versus-host disease (GVHD). This condition occurs when the donor stem cells that make up your new immune system see your body's tissues and organs as something foreign and attack them.

Many people who have an allogeneic transplant get GVHD at some point. The risk of GVHD is a bit greater if the stem cells come from an unrelated donor, but it can happen to anyone who gets a bone marrow transplant from a donor.

GVHD may happen at any time after your transplant. However, it's more common after your bone marrow has started to make healthy cells.

There are two kinds of GVHD: acute and chronic. Acute GVHD usually happens earlier, during the first months after your transplant. It typically affects your skin, digestive tract or liver. Chronic GVHD typically develops later and can affect many organs.

Chronic GVHD signs and symptoms include:

You'll undergo a series of tests and procedures to assess your general health and the status of your condition, and to ensure that you're physically prepared for the transplant. The evaluation may take several days or more.

In addition, a surgeon or radiologist will implant a long thin tube (intravenous catheter) into a large vein in your chest or neck. The catheter, often called a central line, usually remains in place for the duration of your treatment. Your transplant team will use the central line to infuse the transplanted stem cells and other medications and blood products into your body.

If a transplant using your own stem cells (autologous transplant) is planned, you'll undergo a procedure called apheresis (af-uh-REE-sis) to collect blood stem cells.

Before apheresis, you'll receive daily injections of growth factor to increase stem cell production and move stem cells into your circulating blood so that they can be collected.

During apheresis, blood is drawn from a vein and circulated through a machine. The machine separates your blood into different parts, including stem cells. These stem cells are collected and frozen for future use in the transplant. The remaining blood is returned to your body.

If a transplant using stem cells from a donor (allogeneic transplant) is planned, you will need a donor. When you have a donor, stem cells are gathered from that person for the transplant. This process is often called a stem cell harvest or bone marrow harvest. Stem cells can come from your donor's blood or bone marrow. Your transplant team decides which is better for you based on your situation.

Another type of allogeneic transplant uses stem cells from the blood of umbilical cords (cord blood transplant). Mothers can choose to donate umbilical cords after their babies' births. The blood from these cords is frozen and stored in a cord blood bank until needed for a bone marrow transplant.

After you complete your pretransplant tests and procedures, you begin a process known as conditioning. During conditioning, you'll undergo chemotherapy and possibly radiation to:

The type of conditioning process you receive depends on a number of factors, including your disease, overall health and the type of transplant planned. You may have both chemotherapy and radiation or just one of these treatments as part of your conditioning treatment.

Side effects of the conditioning process can include:

You may be able to take medications or other measures to reduce such side effects.

Based on your age and health history, your doctor may recommend lower doses or different types of chemotherapy or radiation for your conditioning treatment. This is called reduced-intensity conditioning.

Reduced-intensity conditioning kills some cancer cells and somewhat suppresses your immune system. Then, the donor's cells are infused into your body. Donor cells replace cells in your bone marrow over time. Immune factors in the donor cells may then fight your cancer cells.

Your bone marrow transplant occurs after you complete the conditioning process. On the day of your transplant, called day zero, stem cells are infused into your body through your central line.

The transplant infusion is painless. You are awake during the procedure.

The transplanted stem cells make their way to your bone marrow, where they begin creating new blood cells. It can take a few weeks for new blood cells to be produced and for your blood counts to begin recovering.

Bone marrow or blood stem cells that have been frozen and thawed contain a preservative that protects the cells. Just before the transplant, you may receive medications to reduce the side effects the preservative may cause. You'll also likely be given IV fluids (hydration) before and after your transplant to help rid your body of the preservative.

Side effects of the preservative may include:

Not everyone experiences side effects from the preservative, and for some people those side effects are minimal.

When the new stem cells enter your body, they begin to travel through your body and to your bone marrow. In time, they multiply and begin to make new, healthy blood cells. This is called engraftment. It usually takes several weeks before the number of blood cells in your body starts to return to normal. In some people, it may take longer.

In the days and weeks after your bone marrow transplant, you'll have blood tests and other tests to monitor your condition. You may need medicine to manage complications, such as nausea and diarrhea.

After your bone marrow transplant, you'll remain under close medical care. If you're experiencing infections or other complications, you may need to stay in the hospital for several days or sometimes longer. Depending on the type of transplant and the risk of complications, you'll need to remain near the hospital for several weeks to months to allow close monitoring.

You may also need periodic transfusions of red blood cells and platelets until your bone marrow begins producing enough of those cells on its own.

You may be at greater risk of infections or other complications for months to years after your transplant.

A bone marrow transplant can cure some diseases and put others into remission. Goals of a bone marrow transplant depend on your individual situation, but usually include controlling or curing your disease, extending your life, and improving your quality of life.

Some people complete bone marrow transplantation with few side effects and complications. Others experience numerous challenging problems, both short and long term. The severity of side effects and the success of the transplant vary from person to person and sometimes can be difficult to predict before the transplant.

It can be discouraging if significant challenges arise during the transplant process. However, it is sometimes helpful to remember that there are many survivors who also experienced some very difficult days during the transplant process but ultimately had successful transplants and have returned to normal activities with a good quality of life.

Explore Mayo Clinic studies testing new treatments, interventions and tests as a means to prevent, detect, treat or manage this disease.

Living with a bone marrow transplant or waiting for a bone marrow transplant can be difficult, and it's normal to have fears and concerns.

Having support from your friends and family can be helpful. Also, you and your family may benefit from joining a support group of people who understand what you're going through and who can provide support. Support groups offer a place for you and your family to share fears, concerns, difficulties and successes with people who have had similar experiences. You may meet people who have already had a transplant or who are waiting for a transplant.

To learn about transplant support groups in your community, ask your transplant team or social worker for information. Also, several support groups are offered at Mayo Clinic in Arizona, Florida and Minnesota.

Mayo Clinic researchers study medications and treatments for people who have had bone marrow transplants, including new medications to help you stay healthy after your bone marrow transplant.

If your bone marrow transplant is using stem cells from a donor (allogeneic transplant), you may be at risk of graft-versus-host disease. This condition occurs when a donor's transplanted stem cells attack the recipient's body. Doctors may prescribe medications to help prevent graft-versus-host disease and reduce your immune system's reaction (immunosuppressive medications).

After your transplant, it will take time for your immune system to recover. You may be given antibiotics to prevent infections. You may also be prescribed antifungal, antibacterial or antiviral medications. Doctors continue to study and develop several new medications, including new antifungal medications, antibacterial medications, antiviral medications and immunosuppressive medications.

After your bone marrow transplant, you may need to adjust your diet to stay healthy and to prevent excessive weight gain. Maintaining a healthy weight can help prevent high blood pressure, high cholesterol and other negative health effects.

Your nutrition specialist (dietitian) and other members of your transplant team will work with you to create a healthy-eating plan that meets your needs and complements your lifestyle. Your dietitian may also give you food suggestions to control side effects of chemotherapy and radiation, such as nausea.

Your dietitian will also provide you with healthy food options and ideas to use in your eating plan. Your dietitian's recommendations may include:

After your bone marrow transplant, you may make exercise and physical activity a regular part of your life to continue to improve your health and fitness. Exercising regularly helps you control your weight, strengthen your bones, increase your endurance, strengthen your muscles and keep your heart healthy.

Your treatment team may work with you to set up a routine exercise program to meet your needs. You may perform exercises daily, such as walking and other activities. As you recover, you can slowly increase your physical activity.

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Bone marrow transplant - Mayo Clinic

What is a Stem Cell Transplant (Bone Marrow Transplant)? | Cancer.Net

A stem cell transplant is a treatment for some types of cancer. For example, you might have one if you have leukemia, multiple myeloma, or some types of lymphoma. Doctors also treat some blood diseases with stem cell transplants.

In the past, patients who needed a stem cell transplant received a bone marrow transplant because the stem cells were collected from the bone marrow. Today, stem cells are usually collected from the blood, instead of the bone marrow. For this reason, they are now more commonly called stem cell transplants.

A part of your bones called bone marrow makes blood cells. Marrow is the soft, spongy tissue inside bones. It contains cells called hematopoietic stem cells (pronounced he-mah-tuh-poy-ET-ick). These cells can turn into several other types of cells. They can turn into more bone marrow cells. Or they can turn into any type of blood cell.

Certain cancers and other diseases keep hematopoietic stem cells from developing normally. If they are not normal, neither are the blood cells that they make. A stem cell transplant gives you new stem cells. The new stem cells can make new, healthy blood cells.

The main types of stem cell transplants and other options are discussed below.

Autologous transplant. Doctors call this an AUTO transplant. This type of stem cell transplant may also be called high-dose chemotherapy with autologous stem cell rescue.

In an AUTO transplant, you get your own stem cells after doctors treat the cancer. First, your health care team collects stem cells from your blood and freezes them. Next, you have powerful chemotherapy, and rarely, radiation therapy. Then, your health care team thaws your frozen stem cells. They put them back in your blood through a tube placed in a vein (IV).

It takes about 24 hours for your stem cells to reach the bone marrow. Then they start to grow, multiply, and help the marrow make healthy blood cells again.

Allogeneic transplantation. Doctors call this an ALLO transplant.

In an ALLO transplant, you get another persons stem cells. It is important to find someone whose bone marrow matches yours. This is because you have certain proteins on your white blood cells called human leukocyte antigens (HLA). The best donor has HLA proteins as much like yours as possible.

Matching proteins make a serious condition called graft-versus-host disease (GVHD) less likely. In GVHD, healthy cells from the transplant attack your cells. A brother or sister may be the best match. But another family member or volunteer might work.

Once you find a donor, you receive chemotherapy with or without radiation therapy. Next, you get the other persons stem cells through a tube placed in a vein (IV). The cells in an ALLO transplant are not typically frozen. So, doctors can give you the cells as soon after chemotherapy or radiation therapy as possible.

There are 2 types of ALLO transplants. The best type for each patient depends his or her age and health and the type of disease being treated.

Ablative, which uses high-dose chemotherapy

Reduced intensity, which uses milder doses of chemotherapy

If your health care team cannot find a matched adult donor, there are other options. Research is ongoing to determine which type of transplant will work best for different patients.

Umbilical cord blood transplant. This may be an option if you cannot find a donor match. Cancer centers around the world use cord blood.

Parent-child transplant and haplotype mismatched transplant. These types of transplants are being used more commonly. The match is 50%, instead of near 100%. Your donor might be a parent, child, brother, or sister.

Your doctor will recommend an AUTO or ALLO transplant based mostly on the disease you have. Other factors include the health of your bone marrow and your age and general health. For example, if you have cancer or other disease in your bone marrow, you will probably have an ALLO transplant. In this situation, doctors do not recommend using your own stem cells.

Choosing a transplant is complicated. You will need help from a doctor who specializes in transplants. So you might need to travel to a center that does many stem cell transplants. Your donor might need to go, too. At the center, you talk with a transplant specialist and have an examination and tests. Before a transplant, you should also think about non-medical factors. These include:

Who can care for you during treatment

How long you will be away from work and family responsibilities

If your insurance pays for the transplant

Who can take you to transplant appointments

Your health care team can help you find answers to these questions.

The information below tells you the main parts of AUTO and ALLO transplants. Your health care team usually does the steps in order. But sometimes certain steps happen in advance, such as collecting stem cells. Ask your doctor what to expect before, during, and after a transplant.

A doctor puts a thin tube called a transplant catheter in a large vein. The tube stays in until after the transplant. Your health care team will collect stem cells through this tube and give chemotherapy and other medications through the tube.

You get injections of a medication to raise your number of white blood cells. White blood cells help your body fight infections.

Your health care team collects stem cells, usually from your blood.

Time: 1 to 2 weeks

Where its done: Clinic or hospital building. You do not need to stay in the hospital overnight.

Time: 5 to 10 days

Where its done: Clinic or hospital. At many transplant centers, patients need to stay in the hospital for the duration of the transplant, usually about 3 weeks. At some centers, patients receive treatment in the clinic and can come in every day.

Time: Each infusion usually takes less than 30 minutes. You may receive more than 1 infusion.

Where its done: Clinic or hospital.

Time: approximately 2 weeks

Where its done: Clinic or hospital. You might be staying in the hospital or you might not.

Time: Varies based on how the stem cells are collected

Where its done: Clinic or hospital

Time: 5 to 7 days

Where its done: Many ALLO transplants are done in the hospital.

Time: 1 day

Where its done: Clinic or hospital.

You take antibiotics and other drugs. This includes medications to prevent graft-versus-host disease. You get blood transfusions through your catheter if needed. Your health care team takes care of any side effects from the transplant.

After the transplant, patients visit the clinic frequently at first and less often over time.

Time: Varies

For an ablative transplant, patients are usually in the hospital for about 4 weeks in total.

For a reduced intensity transplant, patients are in the hospital or visit the clinic daily for about 1 week.

The words successful transplant might mean different things to you, your family, and your doctor. Below are 2 ways to measure transplant success.

Your blood counts are back to safe levels. A blood count is the number of red cells, white cells, and platelets in your blood. A transplant makes these numbers very low for 1 to 2 weeks. This causes risks of:

Infection from low numbers of white cells, which fight infections

Bleeding from low numbers of platelets, which stop bleeding

Tiredness from low numbers of red cells, which carry oxygen

Doctors lower these risks by giving blood and platelet transfusions after a transplant. You also take antibiotics to help prevent infections. When the new stem cells multiply, they make more blood cells. Then your blood counts improve. This is one way to know if a transplant is a success.

It controls your cancer. Doctors do stem cell transplants with the goal of curing disease. A cure may be possible for some cancers, such as some types of leukemia and lymphoma. For other patients, remission is the best result. Remission is having no signs or symptoms of cancer. After a transplant, you need to see your doctor and have tests to watch for any signs of cancer or complications from the transplant.

Talking often with the doctor is important. It gives you information to make health care decisions. The questions below may help you learn more about stem cell transplant. You can also ask other questions that are important to you.

Which type of stem cell transplant would you recommend? Why?

If I will have an ALLO transplant, how will we find a donor? What is the chance of a good match?

What type of treatment will I have before the transplant? Will radiation therapy be used?

How long will my treatment take? How long will I stay in the hospital?

How will a transplant affect my life? Can I work? Can I exercise and do regular activities?

How will we know if the transplant works?

What if the transplant doesnt work? What if the cancer comes back?

What are the side effects? This includes short-term, such as during treatment and shortly after. It also includes long-term, such as years later.

What tests will I need later? How often will I need them?

If I am worried about managing the costs of treatment, who can help me with these concerns?

Bone Marrow Aspiration and Biopsy

Making Decisions About Cancer Treatment

Donating Blood and Platelets

Donating Umbilical Cord Blood

Explore BMT

Be the Match: National Marrow Donor Program

Blood & Marrow Transplant Information Network

U.S. Department of Health and Human Services: Understanding Transplantation as a Treatment Option

National Bone Marrow Transplant Link

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What is a Stem Cell Transplant (Bone Marrow Transplant)? | Cancer.Net

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Bone marrow | anatomy | Britannica.com

Bone marrow, also called myeloid tissue, soft, gelatinous tissue that fills the cavities of the bones. Bone marrow is either red or yellow, depending upon the preponderance of hematopoietic (red) or fatty (yellow) tissue. In humans the red bone marrow forms all of the blood cells with the exception of the lymphocytes, which are produced in the marrow and reach their mature form in the lymphoid organs. Red bone marrow also contributes, along with the liver and spleen, to the destruction of old red blood cells. Yellow bone marrow serves primarily as a storehouse for fats but may be converted to red marrow under certain conditions, such as severe blood loss or fever. At birth and until about the age of seven, all human marrow is red, as the need for new blood formation is high. Thereafter, fat tissue gradually replaces the red marrow, which in adults is found only in the vertebrae, hips, breastbone, ribs, and skull and at the ends of the long bones of the arm and leg; other cancellous, or spongy, bones and the central cavities of the long bones are filled with yellow marrow.

Red marrow consists of a delicate, highly vascular fibrous tissue containing stem cells, which differentiate into various blood cells. Stem cells first become precursors, or blast cells, of various kinds; normoblasts give rise to the red blood cells (erythrocytes), and myeloblasts become the granulocytes, a type of white blood cell (leukocyte). Platelets, small blood cell fragments involved in clotting, form from giant marrow cells called megakaryocytes. The new blood cells are released into the sinusoids, large thin-walled vessels that drain into the veins of the bone. In mammals, blood formation in adults takes place predominantly in the marrow. In lower vertebrates a number of other tissues may also produce blood cells, including the liver and the spleen.

Because the white blood cells produced in the bone marrow are involved in the bodys immune defenses, marrow transplants have been used to treat certain types of immune deficiency and hematological disorders, especially leukemia. The sensitivity of marrow to damage by radiation therapy and some anticancer drugs accounts for the tendency of these treatments to impair immunity and blood production.

Examination of the bone marrow is helpful in diagnosing certain diseases, especially those related to blood and blood-forming organs, because it provides information on iron stores and blood production. Bone marrow aspiration, the direct removal of a small amount (about 1 ml) of bone marrow, is accomplished by suction through a hollow needle. The needle is usually inserted into the hip or sternum (breastbone) in adults and into the upper part of the tibia (the larger bone of the lower leg) in children. The necessity for a bone marrow aspiration is ordinarily based on previous blood studies and is particularly useful in providing information on various stages of immature blood cells. Disorders in which bone marrow examination is of special diagnostic value include leukemia, multiple myeloma, Gaucher disease, unusual cases of anemia, and other hematological diseases.

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Bone marrow | anatomy | Britannica.com

Bone Marrow Transplantation | Bone Marrow Transplant …

Bone marrow is the spongy tissue inside some of your bones, such as your hip and thigh bones. It contains immature cells, called stem cells. The stem cells can develop into red blood cells, which carry oxygen throughout the body, white blood cells, which fight infections, and platelets, which help the blood to clot.

A bone marrow transplant is a procedure that replaces a person's faulty bone marrow stem cells. Doctors use these transplants to treat people with certain diseases, such as

Before you have a transplant, you need to get high doses of chemotherapy and possibly radiation. This destroys the faulty stem cells in your bone marrow. It also suppresses your body's immune system so that it won't attack the new stem cells after the transplant.

In some cases, you can donate your own bone marrow stem cells in advance. The cells are saved and then used later on. Or you can get cells from a donor. The donor might be a family member or unrelated person.

Bone marrow transplantation has serious risks. Some complications can be life-threatening. But for some people, it is the best hope for a cure or a longer life.

NIH: National Heart, Lung, and Blood Institute

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Bone Marrow Transplantation | Bone Marrow Transplant ...

Bone Marrow and Stem Cell Transplant | Cook Children’s

Certain diseases and treatments can deplete a child's healthy stem cells. Sometimes the body needs help to replenish those cells. When this happens, your child may require a very complex process called a stem cell or bone marrow transplant.

Since 1986, Cook Children's Bone Marrow and Stem Cell Transplant program has performed more than 1,000 transplants in children with cancer, blood disorders or inherited conditions. That's what makes this program one of the more diverse and experienced pediatric transplant programs in the Southwest.

Cook Children's is a member of:

Over the last three years, 30 to 40 transplants were performed every year for a variety of diseases, with leukemia being the most common primary diagnosis.

The goal of the program is to provide a stem cell or marrow transplant to any child who needs one and to improve the outcomes for these patients who do not have better therapy options. We work to achieve this goal through excellent clinical care from several services within Cook Children's, quality initiatives and ongoing comparison of our processes and performance against large academic transplant centers and international data.

Common referral diagnoses:

Stem cells are cells in the body that have the potential to turn into anything, such as a skin cell, a liver cell, a brain cell, or a blood cell. Stem cells that turn into blood cells are called hematopoietic stem cells. These cells are capable of developing into the three types of blood cells:

Stem cells may come from the patient or from a donor. Stem cells that come from a patient may come from their own cord blood cells if they were harvested from the mother's placenta immediately after the child was born and frozen for later use. Stem cells may also be harvested and frozen before the child or teen undergoes treatment. These stem cells are thawed and put back into the patient's body after treatment is complete.

Donor stem cells come from a compatible family member or through a match from a national registry of donors. Depending on the particular needs of your child, one or all three types of a donor's stem cells will be harvested:

While all three types can replenish a patient's blood and bone marrow cells, there are advantages and disadvantages to each. The doctor will discuss these issues and suggest the best type of stem cell for your child's illness.

If your child has been diagnosed, you probably have lots of questions. We can help. If you would like to schedule an appointment, refer a patient or speak to our staff, please call our offices at 682-885-4007.

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Bone Marrow and Stem Cell Transplant | Cook Children's

Bone marrow transplant – About – Mayo Clinic

Overview

A bone marrow transplant is a procedure that infuses healthy blood stem cells into your body to replace your damaged or diseased bone marrow. A bone marrow transplant is also called a stem cell transplant.

A bone marrow transplant may be necessary if your bone marrow stops working and doesn't produce enough healthy blood cells.

Bone marrow transplants may use cells from your own body (autologous transplant) or from a donor (allogeneic transplant).

Mayo Clinic's approach

A bone marrow transplant may be used to:

Bone marrow transplants can benefit people with a variety of both cancerous (malignant) and noncancerous (benign) diseases, including:

Bone marrow is the spongy tissue inside some bones. Its job is to produce blood cells. If your bone marrow isn't functioning properly because of cancer or another disease, you may receive a stem cell transplant.

To prepare for a stem cell transplant, you receive chemotherapy to kill the diseased cells and malfunctioning bone marrow. Then, transplanted blood stem cells are put into your bloodstream. The transplanted stem cells find their way to your marrow, where ideally they begin producing new, healthy blood cells.

A bone marrow transplant poses many risks of complications, some potentially fatal.

The risk can depend on many factors, including the type of disease or condition, the type of transplant, and the age and health of the person receiving the transplant.

Although some people experience minimal problems with a bone marrow transplant, others may develop complications that may require treatment or hospitalization. Some complications could even be life-threatening.

Complications that can arise with a bone marrow transplant include:

Your doctor can explain your risk of complications from a bone marrow transplant. Together you can weigh the risks and benefits to decide whether a bone marrow transplant is right for you.

If you receive a transplant that uses stem cells from a donor (allogeneic transplant), you may be at risk of developing graft-versus-host disease (GVHD). This condition occurs when the donor stem cells that make up your new immune system see your body's tissues and organs as something foreign and attack them.

Many people who have an allogeneic transplant get GVHD at some point. The risk of GVHD is a bit greater if the stem cells come from an unrelated donor, but it can happen to anyone who gets a bone marrow transplant from a donor.

GVHD may happen at any time after your transplant. However, it's more common after your bone marrow has started to make healthy cells.

There are two kinds of GVHD: acute and chronic. Acute GVHD usually happens earlier, during the first months after your transplant. It typically affects your skin, digestive tract or liver. Chronic GVHD typically develops later and can affect many organs.

Chronic GVHD signs and symptoms include:

You'll undergo a series of tests and procedures to assess your general health and the status of your condition, and to ensure that you're physically prepared for the transplant. The evaluation may take several days or more.

In addition, a surgeon or radiologist will implant a long thin tube (intravenous catheter) into a large vein in your chest or neck. The catheter, often called a central line, usually remains in place for the duration of your treatment. Your transplant team will use the central line to infuse the transplanted stem cells and other medications and blood products into your body.

If a transplant using your own stem cells (autologous transplant) is planned, you'll undergo a procedure called apheresis (af-uh-REE-sis) to collect blood stem cells.

Before apheresis, you'll receive daily injections of growth factor to increase stem cell production and move stem cells into your circulating blood so that they can be collected.

During apheresis, blood is drawn from a vein and circulated through a machine. The machine separates your blood into different parts, including stem cells. These stem cells are collected and frozen for future use in the transplant. The remaining blood is returned to your body.

If a transplant using stem cells from a donor (allogeneic transplant) is planned, you will need a donor. When you have a donor, stem cells are gathered from that person for the transplant. This process is often called a stem cell harvest or bone marrow harvest. Stem cells can come from your donor's blood or bone marrow. Your transplant team decides which is better for you based on your situation.

Another type of allogeneic transplant uses stem cells from the blood of umbilical cords (cord blood transplant). Mothers can choose to donate umbilical cords after their babies' births. The blood from these cords is frozen and stored in a cord blood bank until needed for a bone marrow transplant.

After you complete your pretransplant tests and procedures, you begin a process known as conditioning. During conditioning, you'll undergo chemotherapy and possibly radiation to:

The type of conditioning process you receive depends on a number of factors, including your disease, overall health and the type of transplant planned. You may have both chemotherapy and radiation or just one of these treatments as part of your conditioning treatment.

Side effects of the conditioning process can include:

You may be able to take medications or other measures to reduce such side effects.

Based on your age and health history, your doctor may recommend lower doses or different types of chemotherapy or radiation for your conditioning treatment. This is called reduced-intensity conditioning.

Reduced-intensity conditioning kills some cancer cells and somewhat suppresses your immune system. Then, the donor's cells are infused into your body. Donor cells replace cells in your bone marrow over time. Immune factors in the donor cells may then fight your cancer cells.

Your bone marrow transplant occurs after you complete the conditioning process. On the day of your transplant, called day zero, stem cells are infused into your body through your central line.

The transplant infusion is painless. You are awake during the procedure.

The transplanted stem cells make their way to your bone marrow, where they begin creating new blood cells. It can take a few weeks for new blood cells to be produced and for your blood counts to begin recovering.

Bone marrow or blood stem cells that have been frozen and thawed contain a preservative that protects the cells. Just before the transplant, you may receive medications to reduce the side effects the preservative may cause. You'll also likely be given IV fluids (hydration) before and after your transplant to help rid your body of the preservative.

Side effects of the preservative may include:

Not everyone experiences side effects from the preservative, and for some people those side effects are minimal.

When the new stem cells enter your body, they begin to travel through your body and to your bone marrow. In time, they multiply and begin to make new, healthy blood cells. This is called engraftment. It usually takes several weeks before the number of blood cells in your body starts to return to normal. In some people, it may take longer.

In the days and weeks after your bone marrow transplant, you'll have blood tests and other tests to monitor your condition. You may need medicine to manage complications, such as nausea and diarrhea.

After your bone marrow transplant, you'll remain under close medical care. If you're experiencing infections or other complications, you may need to stay in the hospital for several days or sometimes longer. Depending on the type of transplant and the risk of complications, you'll need to remain near the hospital for several weeks to months to allow close monitoring.

You may also need periodic transfusions of red blood cells and platelets until your bone marrow begins producing enough of those cells on its own.

You may be at greater risk of infections or other complications for months to years after your transplant.

A bone marrow transplant can cure some diseases and put others into remission. Goals of a bone marrow transplant depend on your individual situation, but usually include controlling or curing your disease, extending your life, and improving your quality of life.

Some people complete bone marrow transplantation with few side effects and complications. Others experience numerous challenging problems, both short and long term. The severity of side effects and the success of the transplant vary from person to person and sometimes can be difficult to predict before the transplant.

It can be discouraging if significant challenges arise during the transplant process. However, it is sometimes helpful to remember that there are many survivors who also experienced some very difficult days during the transplant process but ultimately had successful transplants and have returned to normal activities with a good quality of life.

Explore Mayo Clinic studies testing new treatments, interventions and tests as a means to prevent, detect, treat or manage this disease.

Living with a bone marrow transplant or waiting for a bone marrow transplant can be difficult, and it's normal to have fears and concerns.

Having support from your friends and family can be helpful. Also, you and your family may benefit from joining a support group of people who understand what you're going through and who can provide support. Support groups offer a place for you and your family to share fears, concerns, difficulties and successes with people who have had similar experiences. You may meet people who have already had a transplant or who are waiting for a transplant.

To learn about transplant support groups in your community, ask your transplant team or social worker for information. Also, several support groups are offered at Mayo Clinic in Arizona, Florida and Minnesota.

Mayo Clinic researchers study medications and treatments for people who have had bone marrow transplants, including new medications to help you stay healthy after your bone marrow transplant.

If your bone marrow transplant is using stem cells from a donor (allogeneic transplant), you may be at risk of graft-versus-host disease. This condition occurs when a donor's transplanted stem cells attack the recipient's body. Doctors may prescribe medications to help prevent graft-versus-host disease and reduce your immune system's reaction (immunosuppressive medications).

After your transplant, it will take time for your immune system to recover. You may be given antibiotics to prevent infections. You may also be prescribed antifungal, antibacterial or antiviral medications. Doctors continue to study and develop several new medications, including new antifungal medications, antibacterial medications, antiviral medications and immunosuppressive medications.

After your bone marrow transplant, you may need to adjust your diet to stay healthy and to prevent excessive weight gain. Maintaining a healthy weight can help prevent high blood pressure, high cholesterol and other negative health effects.

Your nutrition specialist (dietitian) and other members of your transplant team will work with you to create a healthy-eating plan that meets your needs and complements your lifestyle. Your dietitian may also give you food suggestions to control side effects of chemotherapy and radiation, such as nausea.

Your dietitian will also provide you with healthy food options and ideas to use in your eating plan. Your dietitian's recommendations may include:

After your bone marrow transplant, you may make exercise and physical activity a regular part of your life to continue to improve your health and fitness. Exercising regularly helps you control your weight, strengthen your bones, increase your endurance, strengthen your muscles and keep your heart healthy.

Your treatment team may work with you to set up a routine exercise program to meet your needs. You may perform exercises daily, such as walking and other activities. As you recover, you can slowly increase your physical activity.

Oct. 13, 2016

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Bone marrow transplant - About - Mayo Clinic

Glossary of Terms | Aplastic Anemia and MDS International …

acute myelogenous leukemia

(uh-KYOOT my-uh-LAH-juh-nuss loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous. This slows production of red blood cells and platelets. Some cases of MDS become AML. But most do not. Also called AML, acute myeloblastic leukemia, acute myelocytic leukemia, acute myeloid leukemia.

A procedure where bone marrow stem cells are taken from a genetically matched donor (a brother, sister, or unrelated donor) and given to the patient through an intravenous (IV) line. In time, donated stem cells start making new, healthy blood cells.

See complementary and alternative medicine.

(an-uh-fuh-LAK-suss) A very severe allergic reaction to a foreign protein, as in a bee sting, or to a medicine. This reaction causes the blood pressure to drop and trouble breathing. Before a patient receives ATG, a treatment for aplastic anemia, a skin test is given to find out if they are likely to develop anaphylaxis. Also known as anaphylactic shock.

An approach to treating bone marrow failure using natural male hormones. Androgen therapy can help the bone marrow make more blood cells. This is an older treatment for bone marrow failure that is rarely used because of the side effects. Scientists are studying these medicines to try to better understand why they work in some cases of acquired and genetic bone marrow failure.

(uh-NEE-mee-uh) A condition in which there is a shortage of red blood cells in the bloodstream. This causes a low red blood cell count. Symptoms of anemia are fatigue and tiredness.

(an-tee-by-AH-tik) A medicine that fights bacterial infections. When a person with bone marrow failure does not have enough neutrophils, the white blood cells that fight infection, antibiotics may help to prevent and fight infection.

(ant-i-ko-AG-yuh-lunt) See blood thinner.

(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells: red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most scientists believe that aplastic anemia happens when the immune system attacks the bone marrow stem cells. Aplastic anemia can be acquired (begin any time in life) or can be hereditary (less common, passed down from parent to child).

Programmed cell death.

(uh-SITE-eez) Extra fluid and swelling in the belly area (abdomen). Also called hydroperitoneum.

Any condition that happens when the immune system attacks the body's own normal tissues by mistake.

A procedure in which some of the patient's own bone marrow stem cells are removed, frozen, and then returned to the through an intravenous (IV) line. In time, the stem cells start making new, healthy blood cells.

Describes one of several ways that a trait or disorder can be inherited, or passed down through families. "Autosomal" means that the mutated, or abnormal, gene is located on one of the numbered, or non-sex, chromosomes. "Dominant" means that only one copy of the mutated gene is enough to cause the disease. Dyskeratosis congenita is a rare cause of bone marrow failure disease. It may have an autosomal dominant, autosomal recessive or x-linked pattern of inheritance.

Describes one of several ways that a trait or disorder can be inherited, or passed down through families. "Autosomal" means that the mutated, or abnormal, gene is located on one of the numbered, or non-sex, chromosomes. "Recessive" means that two copies of a mutated gene must be present to cause the disease. Dyskeratosis congenita is a rare cause of bone marrow failure. It may have an autosomal dominant, autosomal recessive or x-linked pattern of inheritance.

The study of a subject to increase knowledge and understanding about it. The goal of basic research in medicine is to better understand disease. In the laboratory, basic research scientists study changes in cells and molecules linked to disease. Basic research helps lead to better ways of diagnosing, treating, and preventing disease. Also called basic science research.

A type of white blood cell that plays a role in allergic reactions.

A chemical that is widely used by the chemical industry in the United States to make plastics, resins, nylon and synthetic fibers. Benzene is found in tobacco smoke, vehicle emissions, and gasoline fumes. Exposure to benzene may increase the risk of developing a bone marrow failure disease. Benzene can affect human health by causing bone marrow stem cells not to work correctly.

(bil-i-ROO-bun) A reddish yellow substance formed when red blood cells break apart. It is found in the bile and in the blood. Yellowing of the skin and eyes can occur with high levels of bilirubin. Also called total bilirubin.

A substance made from a living system, such as a virus, and used to prevent or treat disease. Biological drugs include antibodies, globulin, interleukins, serum, and vaccines. Also called a biologic or biological drug.

A young white blood cell. The number of blast cells in the bone marrow helps define how severe MDS is in a person. When 20 out of 100 cells in the bone marrow are blasts, this is considered acute myeloid leukemia.

See Blast Cells.

A mass of blood that forms when platelets stick together. Harmful blood clots are more likely to happen in PNH. The term thrombus describes a blood clot that develops and attaches to a blood vessel. The term embolus describes a blood clot or other foreign matter that gets into the bloodstream and gets stuck in a blood vessel.

A medicine used to stop blood clots from forming. Blood thinners can be used to treat or prevent clots. Some common blood thinners are enoxaprin (Lovenox), heparin (Calciparine or Liquaemin), and warfarin (Coumadin). Also called and anticoagulant or thrombopoiesis inhibitor.

A procedure in which whole blood or one of its components is given to a person through an intravenous (IV) line into the bloodstream. A red blood cell transfusion or a platelet transfuson can help some patients with low blood counts.

The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.

A medical procedure to remove of a small amount of liquid bone marrow through a needle inserted into the back of the hip. The liquid bone marrow is examined for abnormalities in cell size, shape, or look. Tests may also be run on the bone marrow cells to look for any genetic abnormalities.

A medical procedure to remove a small piece of solid bone marrow using a needle that goes into the marrow of the hip bone. The solid bone marrow is examined for cell abnormalities, the number of different cells and checked for scaring of the bone marrow.

A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be hereditary (less common, passed down from parent to child).

A procedure where bone marrow stem cells are collected from marrow inside the donor's hipbone and given to the patient through an intravenous (IV) line. In time, donated stem cells start making new, healthy blood cells.

(bud-kee-AR-ee SIN-drome) A blood clot in the major vein that leaves the liver (hepatic vein). The liver and the spleen may become enlarged. Budd-Chiari syndrome can occur in PNH.

How much of the bone marrow volume is occupied by various types of blood cells.

(kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People with high-risk or intermediate-2 risk myelodysplastic syndrome (MDS) may be given chemotherapy to kill bone marrow cells that have an abnormal size, shape, or look. Chemotherapy hurts healthy cells along with abnormal cells. If chemotherapy works in controlling abnormal cells, then relatively normal blood cells will start to grow again. Low-dose chemotherapy agents include: cytarabine (Ara-C) and hydroxyurea (Hydrea). High-dose chemotherapy agents include: daunorubicin (Cerubidine), idarubicin (Idamycin), and mitoxanrone (Novantrone).

The part of the cell that contains our DNA or genetic code.

A medical condition that lasts a long time. A chronic illness can affect a person's lifestyle, ability to work, physical abilities and independence.

A person who gives advice, or counsel, to people who are coping with long-term illness. A chronic illness counselor helps people understand their abilities and limitations, cope with the stress, pain, and fatigue associated with long-term illness. A chronic illness counselor can often be located by contacting a local hospital.

A type of research that involves individual persons or a group of people. There are three types of clinical research. Patient-oriented research includes clinical trials which test how a drug, medical device, or treatment approach works in people. Epidemiology or behavioral studies look at the patterns and causes of disease in groups of people. Outcomes and health services research seeks to find the most effective treatments and health services.

A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a disease or health problem. Quality of life (supportive care) trials study ways to improve the comfort of people with chronic illness. Prevention trials look for better ways to prevent disease in people who have never had the disease.

Trials are in four phases: Phase I tests a new drug or treatment in a small group to see if it is safe. Phase II expands the study to a larger group of people to find out if it works. Phase III expands the study to an even larger group of people to compare it to the standard treatment for the disease; and Phase IV takes place after the drug or treatment has been licensed and marketed to find out the long-term impact of the new treatment.

To make copies. Bone marrow stem cells clone themselves all the time. The cloned stem cells eventually become mature blood cells that leave the bone marrow and enter the bloodstream.

To thicken. Normal blood platelets cause the blood to coagulate and stop bleeding.

A group of proteins that move freely in the bloodstream. These proteins support (complement) the work of white blood cells by fighting infections.

A medical approach that is not currently part of standard practice. Complementary medicine is used along with standard medicine. Alternative medicine is used in place of standard medicine. Example of CAM therapies are acupuncture, chiropractic, homeopathic, and herbal medicines. There is no complementary or alternative therapy that effectively treats bone marrow failure. Some CAM therapies may even hinder the effectiveness of standard medical care. Patients should talk with their doctor if they are currently using or considering using a complementary or alternative therapy.

A group of tests performed on a small amount of blood. The CBC measures the number of each blood cell type, the size of the red blood cells, the total amount of hemoglobin, and the fraction of the blood made up of red blood cells. Also called a CBC.

A procedure where umbillical cord stem cells are given to the patient through an intravenous (IV) line. Stem cells are collected from an umbilical cord right after the birth of a baby. They are kept frozen until needed. In time, donated stem cells given to the patient begin making new, healthy blood cells.

An imaging technique using x-ray technology and computerization to create a three-dimentional image of a body part. Also called a CT scan, it can be used to locate a blood clot in the body.

A response to treatment indicating that no sign of abnormal chromosomes are found. When a test is done on a patient with 5q deletion MDS, and there are no signs of an abnormal chromosome 5, then that patient has achieved a cytogenetic remission. Also called cytegenetic response.

(sie-toe-juh-NEH-tiks) The study of chromosomes (DNA), the part of the cell that contains genetic information. Some cytogenetic abnormalities are linked to different forms of myelodysplastic syndromes (MDS).

(sie-tuh-PEE-nee-uh) A shortage of one or more blood cell types. Also called a low blood count.

(sie-tuh-TOK-sik) A medicine that kills certain cells. Chemotherapy for MDS patients often involves the use of cytotoxic agents.

A test that helps doctors find out if a person has a problem with blood clotting.

(di-NO-vo) Brand new, referring to the first time something occurs. MDS that is untreated or that has no known cause is called de novo MDS.

The death of part of the intestine. This can happen if the blood supply in the intestine is cut off, for example, from a blood clot in the abdomen. Also called intestinal necrosis, ischemic bowel, dead gut.

A rare form of pure red cell aplasia that can be passed down from parent to child. Diamond-Blackfan anemia (DBA) is characterized by low red blood cell counts detected in the first year of life. Some people with DBA have physical abnormalities such as small head size, low frontal hairline, wide-set eyes, low-set ears. Genetic testing is used to diagnose DBA.

Vitamins, minerals, herbs and other substances meant to improve your nutritional intake. Dietary supplements are taken by mouth in the form of a pill, capsule, tablet or liquid.

To become distinct or specialized. In the bone marrow, young parent cells (stem cells) develop, or differentiate, into specific types of blood cells (red cells, white cells, platelets).

The gene that always expresses itself over a recessive gene. A person with a dominant gene for a disease has the symptoms of the disease. They can pass the disease on to children.

An inherited disease that may lead to bone marrow failure.

Refers to how well a graft (donor cells) is accepted by the host (the patient) after a bone marrow or stem cell transplant. Several factors contribute to better engraftment: physical condition of the patient, how severe the disease is, type of donor available, age of patient. Successful engraftment results in new bone marrow that produces healthy blood cells.

A type of white blood cell that kills parasites and plays a role in allergic reactions.

The study of patterns and causes of disease in groups of people. Epidemiology researchers study how many people have a disease, how many new cases are diagnosed each year, where patients are located, and environmental or other factors that influence disease.

(i-RITH-ruh-site) See red blood cell.

(i-rith-row-POY-uh-tun) A protein made by the kidneys. Erythropoietin, also called EPO, is created in response to low oxygen levels in the body (anemia). EPO causes the bone marrow to make more red blood cells. A shortage of EPO can also cause anemia.

A medicine used to help the bone marrow make more red blood cells. Epoetin alfa (Epogen, Procrit) and darbepoetin alfa (Aranesp) are erythropoietin-stimulating agents that can help boost the red blood cell count of some bone marrow failure patients. Also called red blood cell growth factor.

A form of estrogen, it is the most potent female hormone. It is also present in males. Estradiol is involved in many body functions beyond the reproductive system. Researchers are investigating the role of estradiol in the treatment of genetic bone marrow failure.

The cause or origin of a disease.

A criteria used for classifying different types of myelodysplastic syndromes (MDS). The FAB (French, American, British) Classification System was developed by a group of French, American and British scientists. This system is based on 2 main factors: the percentage of blast cells in bone marrow, and the percentage of blast cells in the bloodstream. The FAB system is somewhat outdated, but is still used by some doctors today. The World Health Organization (WHO) Classification System has largely replaced the FAB Classification System.

A rare inherited disorder that happens when the bone marrow does not make enough blood cells: red cells, white cells, and platelets. Fanconi anemia is diagnosed early in life. People with Fanconi anemia have a high likelihood of developing cancer. Genetic testing is used to diagnose Fanconi anemia.

(FER-i-tin) A protein inside of cells that stores iron for later use by your body. Sometimes ferritin is released into the blood. The ferritin level in the blood is called serum ferritin.

(FER-i-tin) A blood test used to monitor how much iron the body is storing for later use.

(fie-BRO-suss) Scarring of tissue. Fibrosis of the bone marrow is an feature seen in some types of unclassified myeldysplastic syndrome (MDS).

See fluorescence in situ hybridization.

(sy-TOM-uh-tree) A laboratory test that gives information about cells, such as size, shape, and percentage of live cells. Flow cytometry is the test doctors use to see if there are any proteins missing from the surface of blood cells. It is the standard test for confirming a diagnosis of paroxysmal nocturnal hemoglobinuria (PNH).

(flor-EH-sense in SIT-tyoo hy-bru-duh-ZAY-shun) An important laboratory test used to help doctors look for chromosomal abnormalities and other genetic mutations. Fluorescence in situ hybridization, also called FISH, directs colored light under a microscope at parts of chromosomes or genes. Missing or rearranged chromosomes are identified using FISH.

(FOE-late) A B-vitamin that is found in fresh or lightly cooked green vegetables. It helps the bone marrow make normal blood cells. Most people get enough folate in their diet. Doctors may have people with paroxysmal nocturnal hemaglobinuria (PNH) take a man-made form of folate called folic acid.

See folate.

A laboratory test that looks at the whether red blood cells break apart too easily when they are placed in mild acid. This test has been used in the past to diagnose paroxysmal nocturnal hemoglobinuria (PNH). Most doctors now use flow cytometry, a more accurate method of testing for PNH. Ham Test is also called acid hemolysin test.

(hi-MA-tuh-crit) A blood test that measures the percentage of the blood made up of red blood cells. This measurement depends on the number of red blood cells and their size. Hematocrit is part of a complete blood count. Also called HCT, packed cell volume, PCV.

(hee-muh-TOL-uh-jist) A doctor who specializes in treating blood diseases and disorders of blood producing organs.

(hi-mat-uh-poy-EE-suss) The process of making blood cells in the bone marrow.

A condition that occurs when the body absorbs and stores too much iron. This leads to a condition called iron overload. In the United States, hemochromatosis is usually caused by a genetic disorder. Organ damage can occur if iron overload is not treated.

A protein in the red blood cells. Hemoglobin picks up oxygen in the lungs and brings it to cells in all parts of the body.

(hee-muh-gloe-buh-NYOOR-ee-uh) The presence of hemoglobin in the urine.

(hi-MOL-uh-suss) The destruction of red blood cells.

See human leukocyte antigen.

A part of the endocrine system that serves as the body's chemical messengers. Hormones move through the bloodstream to transfer information and instruction from one set of cells to another.

(LEW-kuh-site ANT-i-jun) One of a group of proteins found on the surface of white blood cells and other cells. These antigens differ from person to person. A human leukocyte antigen test is done before a stem cell transplant to closely match a donor and a recipient. Also called HLA.

A condition in which there are too many cells, for example, within the bone marrow. Patients with leukemia have hypercellular bone marrow filled with to many immature white blood cells.

A condition in which there are too few cells, for example, within the bone marrow. Patients with aplastic anemia have hypocellular bone marrow.

Usually refers to any condition with no known cause.

(i-myoo-no-KOM-pruh-mized) Occurs when the immune system is not functioning properly, leaving the patient open to infection. A person can be immunocompromised due to low white blood cell count or due to some medicines. Also called immune compromised.

(i-myoo-no-suh-PREH-siv) Drugs that lower the body's immune response and allow the bone marrow stem cells to grow and make new blood cells. ATG (antithymocyte globulin) or ALG (antilymphocyte globulin) with cyclosporine are used to treat bone marrow failure in aplastic anemia. Immunosuppressive drugs may help some patients with myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH).

A committee that makes sure a clinical trial is safe for patients in the study. Each medical center, hospital, or research facility doing clinical trials must have an active Institutional Review Board (IRB). Each IRB is made up of a diverse group of doctors, faculty, staff and students at a specific institution.

A system that turns patient data into a score. The score tells how quickly a myelodysplastic syndrome (MDS) case is progressing and helps predict what may happen with the patient's MDS in the future. Also called IPSS.

A method of getting fluids or medicines directly into the bloodstream over a period of time. Also called IV infusion.

A new drug, antibiotic drug, or biological drug that is used in a clinical trial. It also includes a biological product used in the laboratory for diagnostic purposes. Also called IND.

(kee-LAY-shun) A drug therapy to remove extra iron from the body. Patients with high blood iron (ferritin) levels may receive iron chelation therapy. The U.S. Food and Drug Administratin (FDA) has approved two iron chelators to treat iron overload in the U.S.: deferasirox, an oral iron chelator, and deferoxamine, a liquid given by injection.

A condition that occurs when too much iron accumulates in the body. Bone marrow failure disease patients who need regular red blood cell transfusions are at risk for iron overload. Organ damage can occur if iron overload is not treated.

(iss-KEE-mee-uh) Occurs when the blood supply to specific organ or part of the body is cut off, causing a localized lack of oxygen.

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bone marrow/stem cell transplant – verywell.com

If you or a loved one will be having a bone marrow transplant or donating stem cells, what does it entail? What are the different types of bone marrow transplants and what is the experience like for both the donor and recipient?

A bone marrow transplant is a procedure in which when special cells (called stem cells) are removed from the bone marrow or peripheral blood, filtered and given back either to the same person or to another person.

Since we now derive most stem cells needed from the blood rather than the bone marrow, a bone marrow transplant is now more commonly referred to as stem cell transplant.

Bone marrow is found in larger bones in the body such as the pelvic bones. This bone marrow is the manufacturing site for stem cells. Stem cells are "pluripotential" meaning that the cells are the precursor cells which can evolve into the different types of blood cells, such as white blood cells, red blood cells, and platelets.

If something is wrong with the bone marrow or the production of blood cells is decreased, a person can become very ill or die. In conditions such as aplastic anemia, the bone marrow stops producing blood cells needed for the body. In diseases such as leukemia, the bone marrow produces abnormal blood cells.

The purpose of a bone marrow transplant is thus to replace cells not being produced or replace unhealthy stem cells with healthy ones.

This can be used to treat or even cure the disease.

In addition for leukemias, lymphomas, and aplastic anemia, stem cell transplants are being evaluated for many disorders, ranging from solid tumors to other non-malignant disorders of the bone marrow, to multiple sclerosis.

There are two primary types of bone marrow transplants, autologous and allogeneic transplants.

The Greek prefix "auto" means "self." In an autologous transplant, the donor is the person who will also receive the transplant. This procedure, also known as a "rescue transplant" involves removing your stem cells and freezing them. You then receive high dose chemotherapy followed by infusion of the thawed out frozen stem cells. It may be used to treat leukemias, lymphomas, or multiple myeloma.

The Greek prefix "allo" means "different" or "other." In an allogeneic bone marrow transplant, the donor is another person who has a genetic tissue type similar to the person needing the transplant. Because tissue types are inherited, similar to hair color or eye color, it is more likely that you will find a suitable donor in a family member, especially a sibling. Unfortunately, this occurs only 25 to 30 percent of the time.

If a family member does not match the recipient, the National Marrow Donor Program Registry database can be searched for an unrelated individual whose tissue type is a close match. It is more likely that a donor who comes from the same racial or ethnic group as the recipient will have the same tissue traits.

Learn more about finding a donor for a stem cell transplant.

Bone marrow cells can be obtained in three primary ways. These include:

The majority of stem cell transplants are done using PBSC collected by apheresis (peripheral blood stem cell transplants.) This method appears to provide better results for both the donor and recipient. There still may be situations in which a traditional bone marrow harvest is done.

Donating stem cells or bone marrow is fairly easy. In most cases, a donation is made using circulating stem cells (PBSC) collected by apheresis. First, the donor receives injections of a medication for several days that causes stem cells to move out of the bone marrow and into the blood. For the stem cell collection, the donor is connected to a machine by a needle inserted in the vein (like for donating blood.) Blood is taken from the vein, filtered by the machine to collect the stem cells, then returned back to the donor through a needle in the other arm. There is almost no need for a recovery time with this procedure.

If stem cells are collected by bone marrow harvest (much less likely,) the donor will go to the operating room and while asleep under anesthesia, a needle will be inserted into either the hip or the breastbone to take out some bone marrow. After awakening, there may be some pain where the needle was inserted.

A bone marrow transplant can be a very challenging procedure for the recipient.

The first step is usually receiving high doses of chemotherapy and/or radiation to eliminate whatever bone marrow is present. For example, with leukemia, it is first important to remove all of the abnormal bone marrow cells.

Once a person's original bone marrow is destroyed, the new stem cells are injected intravenously, similar to a blood transfusion. The stem cells then find their way to the bone and start to grow and produce more cells (called engraftment.)

There are many potential complications. The most critical time is usually when the bone marrow is destroyed so that few blood cells remain. Destruction of the bone marrow results in greatly reduced numbers of all of the types of blood cells (pancytopenia.) Without white blood cells there is a serious risk of infection, and infection precautions are used in the hospital (isolation.) Low levels of red blood cells (anemia) often require blood transfusions while waiting for the new stem cells to begin growing. Low levels of platelets (thrombocytopenia) in the blood can lead to internal bleeding.

A common complication affecting 40 to 80 percent of recipients is graft versus host disease. This occurs when white blood cells (T cells) in the donated cells (graft) attack tissues in the recipient (the host,) and can be life-threatening.

An alternative approach referred to as a non-myeloablative bone marrow transplant or "mini-bone marrow transplant" is somewhat different. In this procedure, lower doses of chemotherapy are given that do not completely wipe out or "ablate" the bone marrow as in a typical bone marrow transplant. This approach may be used for someone who is older or otherwise might not tolerate the traditional procedure. In this case, the transplant works differently to treat the disease as well. Instead of replacing the bone marrow, the donated marrow can attack cancerous cells left in the body in a process referred to as "graft versus malignancy."

If you'd like to become a volunteer donor, the process is straightforward and simple. Anyone between the ages of 18 and 60 and in good health can become a donor. There is a form to fill out and a blood sample to give; you can find all the information you need at the National Marrow Donor Program Web site. You can join a donor drive in your area or go to a local Donor Center to have the blood test done.

When a person volunteers to be a donor, his or her particular blood tissue traits, as determined by a special blood test (histocompatibility antigen test,) are recorded in the Registry. This "tissue typing" is different from a person's A, B, or O blood type. The Registry record also contains contact information for the donor, should a tissue type match be made.

Bone marrow transplants can be either autologous (from yourself) or allogeneic (from another person.) Stem cells are obtained either from peripheral blood, a bone marrow harvest or from cord blood that is saved at birth.

For a donor, the process is relatively easy. For the recipient, it can be a long and difficult process, especially when high doses of chemotherapy are needed to eliminate bone marrow. Complications are common and can include infections, bleeding, and graft versus host disease among others.

That said, bone marrow transplants can treat and even cure some diseases which had previously been almost uniformly fatal. While finding a donor was more challenging in the past, the National Marrow Donor Program has expanded such that many people without a compatible family member are now able to have a bone marrow/stem cell transplant.

Sources:

American Society of Clinical Oncology. Cancer.Net. What is a Stem Cell Transplant (Bone Marrow Transplant)? Updated 01/16. http://www.cancer.net/navigating-cancer-care/how-cancer-treated/bone-marrowstem-cell-transplantation/what-stem-cell-transplant-bone-marrow-transplant

U.S. National Library of Medicine. MedlinePlus. Bone Marrow Transplant. Updated 10/03/17. https://medlineplus.gov/ency/article/003009.htm

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