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Can stem cells cure thalassaemia?

Q: My 15-month-old daughter is suffering from beta-Thalassaemia Major/Intermedia. Currently, her haemoglobin level is in the range of 8-9 and she has not been transfused as yet. I am thinking of stem cell transplantation. Though I have not stored cord blood for my daughter but I have a cousin who has stored cord blood for his daughter at Life Cells Chennai. How do I proceed further with HLA testing? Which are the centres in Delhi where I can get HLA matching for my daughter? Is it true that stem cell transplantation can be done within 3-7 years of age? Please suggest how I can find a donor for my daughter.

A:Thalassaemias are a group of genetic diseases caused by the production of abnormal haemoglobin. Haemoglobin comprises two (beta)-globin and two (alpha)-globin chains, their production being governed by 2 beta and 4 alpha genes respectively. In beta-thalassaemia, the production of beta-chain is abnormal while in alpha-thal it is the alpha-chain. When only one beta-globin gene is affected (thal minor or trait or carrier or heterozygous), it results in mild anaemia not requiring treatment but the involvement of both genes results in severe disease (thalassaemia major or intermedia) requiring regular treatment and follow-up from infancy. Stem cell transplant (SCT) is the only known curative treatment for thalassaemia but is done only in selected patients as the presence of hepatomegaly, portal fibrosis and ineffective chelation prior to transplant, all affect the outcome. The event-free survival rate for patients who have all three features is 59%, compared to 90% for those who lack all 3. Even though blood transfusion is not required after a successful transplant, some patients need continued chelation therapy to remove excess iron. It is a difficult choice to make between standard therapy and SCT for parents of patients with severe thalassaemia as the 15-year cardiac disease-free survival rate for patients receiving standard therapy exceeds 90% and is similar for those without risk factors. The long-term outcome for transplant patients, including fertility, is not known but the cost of long-term standard therapy is higher than the cost of transplant. The possibility of developing cancer after SCT should also be considered. SCT is being done at several centres in India but the largest series is from Christian Medical College, Vellore, Tamil Nadu. In Delhi you need to contact the Department of Haematology at I.R.C.H., All India Institute of Medical Sciences.


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