The Results of Patients with b Thalassemia Major Undergoing Peripheral Blood Stem Cell From Alternative Donor: New Conditioning Regimen for Thalassemia

Abstract 3034 Background: Hematopoietic stem cell transplantation (HSCT) remains the only potentially curative treatment for thalassemia patients, However, most candidates for HSCT do not have a suitable family donor. In order to evaluate whether HSCT from an HLA–well -matched alternative donor can offer a probability of cure comparable to that of HSCT from sibling donor. The new conditioning regimen and peripheral blood stem cell transplant (PBSCT) from alternative donor (NF-08-thalassemia protocol) were used for b thalassemia patients. The improved outcome was summarized here. Objective: To evaluate the overall survival (OS), thalassemia-free survival (TFS), rejection (RE) and transplant related mortality (TRM) of NF-08-thalassemia protocol. Patients and Methods: Sixty-one thalassemia patients, 42 male and 19 female (median age, 7 years; age range, 3–15 years), were transplanted from 56 (32 patients were 8/8 and 24 patients 7/8 locus matched at HLA-A, B, Cw and DRB1) unrelated and 5 (1 patient 8/8 and 4 patients 7/8 antigen matched) parents donors from Oct.2008 to Jan.2011(median follow-up time, 14 months; range, 6–31months). NF-08-thalassemia protocol included Cyclosphosphamide (d-10 to d-9), Busulfan (d-7 to d-5), Thiotepa (d-4), Fludarabine (d-8 to d-4) and ATG (d-3 to d-1). PBSCs from unrelated (56 cases) and parents (5 cases) donor were used as only source of stem cells in all patients. GVHD prophylaxis included Cs A, MMF and sMTX. Results: OS, DFS and TRM in the cohort of 61 patients were 91.8%, 90.2% and 8.2%, respectively. Only one rejected his graft two months after transplantation. Acute GVHD III∼IV was 8.2% (5 patients) and none suffered from extensive chronic GVHD. Three patients died of acute GVHD and two died of the infection after transplantation. Discussion: In comparing this alternative donor HSCT with sibling donor HSCT (30 patients, using the same protocol, meanwhile), OS, TFS, RE and TRM were 91.8% vs. 85.9% (P=0.401), 90.2% vs. 79.2% (p=0.147), 1.6% vs. 6.9% (p=0.197) and 8.2%vs.14.1%, respectively. No significant difference was found. Summary: The results of alternative donor HSCT for b thalassemia patients are comparable with sibling donor transplant with using NF-08-thalassemia protocol. Disclosures: Li: Qi fa Liu: Workmates.