MUMBAI: Three-year-old Kinaya Shah was diagnosed with thalassemia at the tender age of three months and has been undergoing regular blood transfusions ever since. The only cure for thalassemia is a bone marrow transplant (BMT), a form of stem cell therapy. Typically, the donor of the stem cells would be a sibling of the patient such that the stem cells of the donor are a near perfect match to those of the patient. The only complication was that Kinaya was a lone child.

So, city doctors in a first used stem cells donated by Kinaya's father - who was only a half or haploidentical match - to cure the child of the blood disorder. "We went to Vellore, Bangalore and Pune but no one was willing to do the transplant without a full match donor," said Kinaya's parents, Aneri and Shripal Shah. They approached Dr Santanu Sen at the Kokilaben Dhirubhai Ambani Hospital, Andheri, in October of 2016, after reading about a similar surgery that he had performed.

While haploidentical bone marrow transplants are carried out to cure leukaemia, it has only been done about half a dozen times for thalassemia in a couple of Indian cities. ``Haploidentical transplants are gradually increasing because of better techniques,'' said Dr Sen.

Dr Sen has completed 36 BMTs in the last two years, of which 12 were haploidentical donors. ``But this is the first time that a haploidentical transplant has been done in western India to cure thalassemia,'' he said.

Chennai-based haematologist Dr Revathy Raja said that there is a 85% chance of cure in thalassemia with a fully matched donor. ``The success rate falls to 70% with a half-match or haploidentical donor. We have hence not started it at our Chennai centre. Hopefully, techniques will further improve in the coming years,'' she said.

In order to perform the surgery, Dr Sen conditioned Kinaya's immune system over three months, with slight chemotherapy, to increase the chances of her body accepting the graft. "We found that her father's stem cells were a 70% match through genetic tests and decided to use them for the transplant. In the case that the graft was rejected we froze a couple of Kinaya's stem cells as insurance. The positive is that children have lower rejection rates for foreign cells as they have barely developed any active immunity," said Dr Sen. "BMT is the most viable treatment to cure thalassemia, the only barrier thus far was the necessity of a full match donor," he added.

However, Vinay Shetty of NGO Think Foundation, which works for thalassemia patients, said that it would be prudent to wait for a statistically significant number of successful halploidentical transplants before recommending it to all patients.

Post the three months of conditioning, stem cells were collected from her father's bone marrow and the transplant was performed on May 10, 2017. After several tests to confirm that the graft was accepted, Kinaya was finally discharged from the hospital on June 13.

"The future of thalassemia treatment probably lies in gene therapy, but at the moment, haploidentical transplants have made BMT much more accessible," said Dr Sen, adding that he has two more cases such as Kinaya lined up. Kinaya is expected to be completely independent of medication and any trace of thalassemia in the coming six months.

What is Thalassemia?

Thalassemia is a genetic blood disorder when the body produces abnormal hemoglobin. Patients require regular blood transplant and well as dietary control to ensure that blood irons level stay suppressed.

Read more here:
In a first, Mumbai doctors use dad's cells to fight blood disorder - Times of India

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