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Summary  Allogeneic haematopoietic stem cell transplantation ( HSCT ) is the only available curative therapy for patients with thalassaemia major. With the progress in human leucocyte antigen ( HLA ) antigen typing technology and supportive care, the outcomes of thalassaemia major have greatly improved in recent years, even in high‐risk patients. However, the problem of finding a suitable donor is still a major obstacle to curing these patients. In recent decades, the lack of available  HSCT  donors has led to the increased use of haploidentical donors ( HD s) for  HSCT  in haematological malignancies. Recently, we explored the effect of  HD HSCT  to eight children with thalassaemia major based on the  FBCA  conditioning regimen (fludarabine, busulphan, cyclophosphamide, antithymocyte globulin), which is usually used in leukaemia patients receiving haploidentical  HSCT  in our centre. So far, all of the transplanted patients have a stable engraftment and are transfusion independent in daily life. This encouraging result has revised our previous conception about haploidentical  HCST  for thalassaemia major and strongly suggests that  HD HSCT  is a feasible and safe method for thalassaemia major patients.

Haploidentical haematopoietic stem cell transplantation for thalassaemia major based on an FBCA conditioning regimen