Peripheral Blood Stem Cell Transplantation in Patients with Beta-Thalassemia Major

From 1996 to 2002, fifty three patients with major beta-thalassemia received allogeneic peripheral blood stem cell trans plantation (PBSCT). Median age was 6 years. Twenty two were class I, 17 class II and 14 class III. All of the donors were HLA-identical. Conditioning regimen for class I and II patients consisted of Cyclophosphamide (CY) 50 mg/kg/day for 4 days + Busulfan (Bu) 3.5 mg/kg for 4 days, while class III patients received 4 mg/kg/day Busulfan for 4 days and 40mg/kg/day Cyclophosphomide for 4 days. G-CSF (Neopogen) 5µ/kg IV was given to donors. Graft Versus Host Disease (GVHD) prophylaxis regimen consisted of Cyclosporin-A (CsA) 3 mg/kg/day plus Methotrexate (MTX) 10 mg/m2 on day+1 and 6 mg/m2 on days +3 and +6. The median time for neutrophil and platelet engraftment was day +16 and day +23 post transplantation, respectively. Chronic GVHD (cGVHD) was observed in 30 patients (56%). Ten patients (18.8%) died. Forty patients are well and transfusion independent. Median time of follow-up was 23 months. Recurrences have been seen at 3 pts, one patient 21 months, the other one 6 months and the last one 8 months after transplantation, who received Donor Lymphocyte infusion (DLI). Event free survival was 72% and overall survival was 80%. In conclusion, we suggest that PBSCT can be considered a safe and effective treatment for children with Beta-thalassemia major and cGVHD is tolerable and manageable in these patients.