Stem cell (bone marrow) transplant

A haematopoietic stem cell transplant (HSCT) — a bone marrow transplant — is the established cure for thalassaemia major. It replaces the faulty blood-forming cells with healthy ones from a donor, so the body starts making its own normal red cells and transfusions are no longer needed.

Results are best with a fully matched sibling donor and when done young, before organs are affected by years of iron (survival and cure rates are high — around 9 in 10 in the best groups). Matched unrelated and, in expert centres, half-matched (haploidentical) donors are options for those without a sibling match.

It’s a major treatment with real risks (rejection, graft-versus-host disease), so it’s carefully weighed for each person. After a successful transplant, stored iron is still removed — often by simply taking off blood (phlebotomy).