减低剂量预处理异基因造血干细胞移植治疗儿童范可尼贫血四例临床观察

OBJECTIVE: To evaluate the efficiency and safety of low intensity conditional regimen for children with Fanconi anemia (FA) receiving allogenic hematopoietic stem cells transplantation (allo-HSCT). METHODS: Four patients diagnosed as Fanconi anemia were enrolled in this study. One patient received HLA-identical sibling donor hematopoietic stem cell transplantation, two patients underwent unrelated donor matched (UD) HSCT, and one patient received unrelated cord blood transplantation. The conditional regimen consisted of Busulfan with low dose of cyclophosphamide. RESULTS: All 4 cases succeeded in allo-HSCT. The median time for neutrophils engraftment was 11(9–15) day, median time to platelets (PLT) engraftment was 12 (8–28) day. One case occurred with grade I of aGVHD, 1 case with hemorrhagic cystitis. No patient happened with hepatic veno-occlusive disease (VOD). CONCLUSION: Low intensity of conditional regimen is efficient and safe which should be recommended for FA patients with HSCT.