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.