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Welcome to the sixth part of a series on Hemato-oncology.
Question: Dr. Chiragbhai, thank you for explaining briefly about role of Stem Cell Transplant/Bone Marrow Transplant in field of Hematology – for malignant hematological disorders and for benign hematological disorders. Last time you told us about transplant in Aplastic Anemia, and other more rare disorders such as PNH, PRCA.
Now, what about role of transplant in Genetic Disorders?

Answer: As we discussed last time, majority of the disorders for transplant are genetic. However, they are generally uncommon compared to malignancies.
Most known example is Thalassemia. Thalassemia is a disease where there is no other cure. Most children in India die by age 10. Those who are cared for well, live to 20s, and rarely in their 30s. And this requires a great deal of effort on part of family and friends, and society overall. Consider the need for regular and safe blood transfusion every month for life, iron chelation, managing complications of transfusion and of iron overload, growth issues, and financial cost of all this. According to one estimate, overall cost of caring for one thalassemia child by age 20, is about 15lakh.

Even though the blood is frequently provided free to patient, someone is paying for the processing and testing of blood. Costs of transport, loss of work for parents and blood donors etc are not calculated.
Hematopoietic Stem Cell Transplant cures about 80% of Thalassemia children. They become transfusion free, no iron overload and after initial treatment period, enjoy an essentially normal life. And, all this at a cost of only about 6-8 lakh. This is much less compared to cost of caring for such a child lifelong without transplant. Unfortunately, this treatment is not widely available, and only recently, more centers in India and state have started offering this option, although not very regularly. There is an urgent need for state funding to support such programs, as large majority of patients cannot afford a one time cost of 6 lakh.

Que: This is nice to hear that Thalassemia can actually be cured, as we have seen two children in our native place die of thalassemia. We were not aware of such an option. What are the requirements for such a transplant?
Ans: Preferable age is between 2-5 years, i.e. before major side effects of iron overload have begun. Less number of transfusions, less iron overload (ferritin levels are generally followed as a measure, with some new tests recently added), lack of hepatomegaly or significant splenomegaly, fully matched sibling give best results. It is possible at higher age as well, but with somewhat less success rate. Newer medicines have led to increased safety in older patients as well.
Dr. Chirag A. Shah; M.D. Oncology/Hematology (USA), 98243 12144, 98988 31496

Diplomate American Board of Oncology and Hematology. Ahmedabad.
Shyam Hem-Onc Clinic. 402 Galaxy, Near Nehrunagar Circle, Ahmedabad.