Bone marrow transplantation for thalassaemia.

Abstract

For all patients with a histocompatibility antigen (HLA) identical donor we are actually using two protocols to whom the patients is assigned. This is based on which class the patients belongs to at the time of bone-marrow transplant and is independent from the patient's age. For 116 patients in Class 1 and for 271 patients in Class 2 prepared for the transplant with busulfan 14 mg/kg, cyclophosphamide 200 mg/kg and cyclosporin alone, the probabilities of survival and of event-free survival are 95% and 90% for Class 1 and 85% and 81% for Class 2. For 125 Class 3 patients prepared for the transplant with busulfan 14 mg/kg, cyclophosphamide reduced to 120-160 mg/kg, cyclosporin and 'short' methotrexate, the probabilities of survival and of event-free survival are 78% and 54%. For 108 adult patients aged between 17 and 35 years, who underwent the transplant after preparation with the same protocol used for the Class 2 or Class 3 patients, the probabilities of survival are 67% and of event-free survival are 63%. Bone marrow transplantation remains the only form of radical treatment of thalassaemia in those patients with an HLA identical donor.