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Breakthrough in thalassemia treatment




New treatment for Thalessemia increases chances of cure 

Mumbai, 23rd June, 2017: In a break- through to manage the worst affected cases of Thalassemia, doctors at Kokilaben Dhirubhai Ambani Hospital (KDAH) are using haploidentical bone marrow transplants- a procedure usually utilized to cure leukaemia.

Half or haploidentical match using parent’s stem cells opens wide range of scope for patient who is a single child or doesn’t have full match in his/her siblings.

Practically, the probability of second child to be a perfect match for thalassemia major sibling is only 25%. The cure available to a Thalassemia patient is a Bone Marrow Transplant. BMT is generally conducted with full match stem cells from a sibling, such that the stem cells of the donor are a near perfect match to those of the patient. However, the major gap in this process is the availability of the match leading to the vast majority of affected kids not getting the right treatment in India.

In this first- of- its- kind treatment in western India, Kinaya Shah, a 3 and a half year old Mumbai resident, underwent bone marrow transplant with her father’s stem cells which were only a half or haploidentical match.

HLA typing is very important in order to find a donor and recipient whose cells most closely match. Because HLA types are genetically determined, the best chance for a match is a donor who is genetically related to the recipient.

Kinaya was diagnosed with Thalassemia when she was 3 months old. After years of blood transfusions and search of treatment in Vellore, Bengaluru and Pune, Kinaya’s parents got in touch with Dr. Santanu Sen at Kokilaben Dhirubhai Ambani Hospital.

Dr. Santanu Sen conditioned Kinaya’s immune system for over three months with chemotherapy to increase the chances of her body accepting the graft. After three months of conditioning, Dr. Sen collected stem cells from bone marrow of Kinaya’s father and performed the transplant on May 10, 2017. “We found her father’s stem cells were a 70% match through genetic tests and decided to use them for the transplant. In case the graft was rejected, we also froze Kinaya’s own stem cells as insurance in case she rejected her father’s stem cells. Haploidentical transplants have made BMT more accessible and dramatically increases the chances of cure for a child,” said Dr Sen, Pediatric BMT Consultant and Oncologist “Though haplo-identical transplants are being done routinely for malignant conditions such as relapsed leukemias and lymphomas, its use in thalassemia opens up a novel avenue in treating the disease. It has been used with good results internationally and by other institutes in India and we wanted to offer it to the right patients in Mumbai”, said Dr Sen. However, he cautions that the therapy may not be applicable to all patients and long term studies are needed to understand the implications of such a transplant. “For majority of patients, blood transfusion and aggressive medical management would still remain as the gold standard of treatment”


The Thalassemia Therapy Unit at KDAH provides a comprehensive treatment option to all patients with Thalassemia with ready availability of Leucoreduced and irradiated blood products for their patients. Patients are seen in the dedicated Thalassemia Clinic where all aspects of their treatment is monitored and regulated.

KDAH has a dedicated Bone Marrow Transplant unit to provide outstanding care and support to paediatric patients who can be helped by transplantation therapy. The program provides transplant as a treatment option for a variety of acquired and congenital disorders of children and young adults including acute and chronic blood conditions like haematopoietic malignancies, anaemias and bleeding disorders; and inherited immune deficiency syndromes and metabolic diseases.

The Bone Marrow Transplant unit has HEPA filters that provide air free of microorganisms within the unit. The plan of the unit is designed to provide the maximum care and prevention of infections. Thalassemia is one of the most common blood disorders in kids in India. Due to the number of such cases, India is even called the Thalassemia capital of the world. There are about 10000-12000 new cases of Thalassemia born every year in India, out of which the majority may not live beyond 20 years. With repeated blood transfusion that the patients need, iron levels in the blood builds up, which can lead to heart or liver failure. Though all children are given medicines called chelators to get rid of the excess iron in the body, at times these may not be effective or enough in all cases.

In Bone Marrow Transplantation, HLA matching helps prevent rejection as well as graft-versus-host disease (GVHD). Immune system recognizes body’s own cells as ‘self,’ as opposed to ‘foreign,’ based on the HLA proteins displayed on the surface of the cells. The major advantage of a BMT is that after a successful transplant, patient will not need any further transfusions and will not need to be on any long term medications.

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