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Welcome to the seventh part of a series on Hemato-oncology.
Question: Dr. Chiragbhai, thank you for explaining briefly about role of Stem Cell Transplant/Bone Marrow Transplant for Thalassemia. As most of our readers know, thalassemia is very common in Gujarat, with over 10,000 patients, and 1000new patients diagnosed every year. So, transplant in this disease is definitely needed.

But Dr. Chiragbhai, can you take stem cells from a brother or sister who is Thalassemia minor?
Answer: Good question. Yes we can take stem cells from a donor who has thalassemia minor. Patient will be changed from thalassemia major to minor, and minor patient lives essentially a normal life. In fact, 4% of population in Gujarat and 13% of Italy is thalassemia minor.
Que: But then every patient does not have a brother or sister with a match, and many families do not go for a second child once their first child is thalassemia major. Can they also get transplant?

Ans: Another good question. Yes they also can nowadays, which was not possible earlier. This has become possible with use of Umbilical Cord Blood-UCB for stem cell transplant. As you know, cord blood banking is now commercially widely available in India, with at least 3 major companies offering services, and more on way.

Cord blood stored at time of birth, has many circulating stem cells. These stem cells have different properties compared to adult stem cells, which is beneficial for transplant. Cord blood has been already used in over 15,000transplants worldwide for various diseases, including many genetic disorders, and hematological malignancies.

Que: Can you tell us more about the UCB transplant?
Ans: One of the most important advantages of UCB stem cells is their “flexibility”. A transplant using adult stem cells is required to be a 6/6 match, and with any mismatch the risks increase manifold. Compared to that UCB has less risk even with 1-2 point mismatch. This allows many more patients to be transplanted. Also, because of this property, they have less immunological reactions, and hence require shorter duration of immune suppression post transplant.

Another major advantage is faster availability compared with a donor (who needs to be found from a registry, consent taken, HLA typed, and tested for other diseases before being selected as suitable to be a donor), since it is stored in a bank after all necessary tests. Also, there is no risk to donor. Time factor is important for many serious diseases where one cannot wait for few months to find a donor. In fact, it is estimated that if 100,000 UCB units are in storage, it can provide a matched unit for every American.

Disadvantage at present is size i.e. number of stem cells are not sufficient for an adult, and a small potential risk for transmitting an unknown genetic disorder. Issue of number is being resolved by use of 2 or 3 UCB units for an adult, at many centers. Also, research is fast growing to believe that in near future less number of stem cells would be sufficient even for an adult, by use of various methods.
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.