I have a young child with thalassemia who needs monthly blood transfusions in order to survive. Is a cure available for him?

Supportive care with monthly blood transfusions and appropriate medical follow-up will not cure your child, but if followed precisely, may allow your child to live up to 40 or 50 years of age with a good quality of life. The main issue is access to appropriate supportive care and blood transfusions. It is particularly important to assure safe blood, preferably not from family members but from volunteer donors. Pre-transfusion hemoglobin should be kept above 9 g/dL. After the initial 15-20 transfusions, iron from transfused red cells starts to accumulate and may cause harm to your child’s body, especially the heart and liver. This iron build-up is evaluated by measuring ferritin blood levels. When ferritin levels rise above 1,000 ng/mL its time to start iron-removing (chelation) therapy. Your child should be followed by a thalassemia center where the doctors will be able to advise you about supportive care and the different tests needed to assure that he or she will remains as healthy as possible.

Bone Marrow Transplantation(BMT) is the only definitive cure for thalassemia and it consists of replacing your child’s faulty bone marrow stem cells, from which red cells originate, with those obtained from a healthy compatible donor. BMT outcomes have constantly imporved to the point that, in experienced centers, children with a compatible family member can currently expect over 95% cure with normalization of health-related quality of life (see blog article).

These answers have been prepared by:
- Dr. Lawrence Faulkner, Cure2Children medical team coordinator
- Dr. Sadaf Khalid, Cure2Children Pakistan branch coordinator