Allogeneic peripheral blood stem cell transplantation in beta-thalassemia.

Six transfusion-dependent beta-thalassemia major patients were treated with allogeneic peripheral blood stem cell (PBSC) transplant. The donors were HLA identical siblings except one donor who was a father with one-antigen mismatch of HLA-B loci. The donors were mobilized with G-CSF and PBSC was infused without manipulation. Engraftment was documented in all patients. Acute graft versus host disease (GvHD) was present in 4 patients but could be controlled with steroid or/and ATG. One patient died of hepatic veno-occlusive disease (HVOD) and survivors were all transfusion independent (ex-thalassemia). Chronic GvHD occurred in one patient. Allogeneic PBSC transplantation could achieve disease-free survival in beta-thalassemia major patients.