Allogeneic hematopoietic stem cell transplantation for β-thalassemia major patients using a reduced-intensity conditioning regimen / 中华器官移植杂志

ABSTRACT

Objective To observe the efficacy and safety of reduced-intensity conditioning regimen used in allogeneic hematopoietic stem cell transplantation (HSCT) for children with β-thalassemia major.Methods We retrospectively analyzed the clinical data of 15 children with β-thalassemia major undergoing allogeneic HSCT with a reduced-intensity conditioning regimen from March 2013 to March 2017.Fifteen patients were diagnosed definitely,and the median age at transplantation was 5 years (range3-6 years),including 11 with HSCT from unrelated donors (UDs),3 of HLA 8/10 matched and 8 of HLA10/10 matched.The remaining 4 patients out of 15 with HSCT were from related donors with HLA matched,3 donors were siblings and 1 was mother.All patients used a reduced-intensity conditioning regimen.The median mononuclear cell (MNC) dose and CD34 positive cell dose were 11.4 × 108/kg (range4.8-20 × 108/kg)and 9.8 × 106/kg (range5.9-27.2 × 106/kg),respectively.Graft-versus-host disease (GVHD) was prevented by cyclosporine A,methotrexate,MMF and ATGf.Results All 15 patients had successful engraftment.Median time to neutrophil and platelet engraftment was 12 days (range9-21 days) and 15 days (range10-25 days) respectively.Two patients developed grades Ⅱ acute GVHD and 4 patients developed chronic GVHD from unrelated donors,while there was no acute GVHD and 1 patient developed chronic GVHD from related donors.No patients suffered from serious transplantation-related complications,such as hepatic veno-occlusive disease (VOD),hemorrhagic cystitis,EB virus reactivation,CMV reactivation and hepatitis C,etc.The median follow-up time was 24 months (range2-48 months).All patients were healthy and became transfusion-independent.Conclusion The reduced-intensity conditioning regimen proved to be safe and effective for children with β-thalassemia major given allogeneic HSCT.