Is stem cell transplant helpful in treating thalassaemia?
Q: I want to know the cost and place for an umbilical cord blood stem cell transplant in a patient with thalassaemia major who is about 1 year old and has had 2-10 blood infusions with a normal size spleen and liver. Also, can stem cell transplant also help a patient who is 6 years old, has thalassaemia and has had about 150 blood transfusions? He also has hepatospleenomegaly, haemosiderosis and joint pains.
A: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 effect the outcome. The event-free survival rate for patients who have all 3 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 but the largest series is from Christian Medical College, Vellore, Tamil Nadu. You can go to the CMC website and contact the Department of Haematology directly