A Novel Conditioning Regimen Improves Outcomes in b-Thalassemia Major Patients Using Unrelated Donor Peripheral Blood Stem Cell Transplantation

Abstract 4514

We used a novel NF-08-TM transplant protocol in 100 consecutive patients with β-thalassemia major (TM), including 66 with allogeneic peripheral blood stem cell transplantation (PBSCT) from unrelated donors (UD) with well-matched human leukocyte antigens (HLAs) and 34 with hematopoietic stem cell transplantation (HSCT) from matched sibling donors (MSD). The median age at transplantation was 6.5 yo (range: 0.6–15 yo), and the ratio of male to female patients was 68:32. The median follow-up time was 24 months (range: 12–39 months). The 3-year overall survival (OS) and TM-free survival (TFS) were 92.4% and 90.9% in the UD-PBSCT group and 88.2% and 82.4% in the MSD-HSCT group. The cumulative incidences of graft rejection (GR) and grade II–IV acute graft-versus-host disease (aGVHD) were 1.5% and 10.6%, respectively, in the UD-PBSCT group and 6.1% and 5.9%, respectively, in the MSD-HSCT group. The cumulative incidence of transplant-related mortality (TRM) was 7.6% in the UD-PBSCT group and 11.8% in the MSD-HSCT group. In conclusion, UD-PBSCTs using the well-tolerated NF-08-TM protocol show similar results to MSD-HSCTs and can be used to treat b thalassemia patients in the absence of MSD.