Biree Andemariam, MD: There are several factors that impact availability of therapeutic options for individuals with sickle cell disease [SCD]. No. 1 is that, until recently, weve had just very few therapeutic options. Until 2017, we only had 1 FDA [Food and Drug Administration]-approved drug for [SCD], that being hydroxyurea. And then in 2017, we had our second drug approved, L-glutamine. Then in November 2019, we had 2 drugs FDA approved: voxelotor and crizanlizumab. Although were living in a time of accelerated discovery and approvals, we have a long way to go. We are embarking on a time when we have a lot of clinical trial investigations of a lot of different therapies that are targeting many different pathophysiological pathways associated with [SCD] and [SCD]-related complications. There are sort of 2 buckets of approaches. [One approach is] to ameliorate the disease to, for example, reduce the amount of pain individuals experience [and] reduce the degree of anemia they live with.

The other bucket is treatments [with] curative intent, such as the different gene therapy strategies. Were looking at disease modification [and] potentially curative options in an investigational fashion right now, which is super exciting. Im hoping that as the years progress, well have an exponential growth of new available treatments. One issue patients contend with is just not having a lot of therapeutic options. But [another issue that] may be more devastating and more important is not having access to physicians who either have the expertise or desire to care for individuals living with [SCD]. Thus, if we dont fix that problem, which is improving access to specialists with knowledge on how to take care of individuals with [SCD], it doesnt matter how many new drugs we bring to the market, because we need [individuals who] are knowledgeable about the disease to be able to prescribe these new therapies [and] monitor patients for complications to prolong their survival. [Thus, this is a] 2-pronged approach to improving access to care for individuals living with [SCD]: new drugs and more specialists.

When I initiate conversations with my patients about their therapeutic optionsI think its important to understand that even though individuals with [SCD] share a common mutation that gives rise to their disease, the phenotypic manifestations of the disease [and] the individual impact of the disease on the 1 particular patient can vary. Its important to take a patient-specific approach when talking about therapies with patients. Thats how I approach my patients. Not everybodys the same, not everybodys had the same shared, lived experience with [SCD]. There are some patients whose major manifestation of disease are painful crises, and thats their focus. Their focus is to reduce the number of pain episodes they experience, to reduce the number of times they end up in an emergency department or [are] hospitalized. For many patients, thats their goal. For some patients, they dont experience as many pain crises, but they have profound fatigue, have had a stroke and are trying to prevent another stroke, or they have complications such as skin ulcers or priapism episodes. There are some patients who have recurrent manifestations of the acute chest syndrome and end up in the [intensive care unit], intubated and fighting for their lives. And they never want to experience that again.

Every patient has a different manifestation of their [SCD], and when I sit down with a particular patient, [I try] to talk about how their [SCD] affects them. We talk about how it affects them clinically and how it affects them in their everyday lives, their functioning, [and] their quality of life. Are they meeting their lifes goals? What would need to be done in terms of their [SCD] treatment to try to get them toward meeting their lifes goals? Luckily, we now have therapies available that do target some of these key manifestations that are problematic or bothersome to patients. We have 3 drugs that have been approved to reduce pain crisis episodes: hydroxyurea, L-glutamine, and crizanlizumab. And we have 1 drug thats been approved that increases the hemoglobin level by decreasing the hemolytic rate. Improving anemia [and] reducing pain episodes are things that are important to patients, and these are the things I discuss with them.

Wally R. Smith, MD: The therapeutic options associated with [SCD] care were quite limited until [approximately] 15 years ago. At the time, we had very little to offer. We had 1 drug, hydroxyurea, which was approved in 1998 after [many] years of study. We had oxygen [as well as] fluids to try to decrease the concentration of hemoglobin S inside the cell and to keep up with losses via the kidney, [as well as] insensible losses. We [also] had palliative care in the form of opioids and other analgesics for patients who came to the hospital. Until [recently], we werent doing many curative therapies [or] bone marrow transplantations, and we had no other drugs to offer. In 2017, that all changed; we got our second drug. In 2019, we got 2 more drugs approved. Now there are 40, if you will, drugs in the pipeline, all intending on getting approved for patients with [SCD]. Now there is gene therapy to add to bone marrow transplantation as a viable therapeutic option under experimental protocol. Thus, now when a patient comes in and says, What can you do for my [SCD]? we answer that completely different than we did 15 years ago. It is now [approximately] an hour-long conversation to walk through those 4 drugs.

They [have] very different mechanisms of actions [and] [adverse] effect profiles. [The] logic about how to decide which drug to take is not very well worked out. There is no such thing as this drug is best for this kind of patient in my mind. We tell them about each of the drugs. We let them make some choices based on their likelihood of taking them, because sometimes taking the drug is just as much of a problem as prescribing the drug, especially if you dont get a benefit immediately. [There is] no problem taking opioids because you get a pain benefit immediately. [There is a] big problem taking hydroxyurea, the first drug thats available, or even taking the newer drugs that have been approved. Thus, we try to explain it to patients. We even talk to them about curative options. Some of them want to go through with it. Some of them do not because all curative options, at this point, require chemotherapy, a long wait, and a lot of hospital visits and patient visits to the doctor. Thats a turnoff for a lot of patients. We dont just go through a single visit to help [patients] decide what to do therapeutically; we take them through the process and give them time to think, then ask them to either call us or come back and let us know what they want to do because these are lifetime commitments they are making.

Transcript edited for clarity

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Approaching Therapy for Patients With SCD - MD Magazine

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