Hou H, Yao Y H, Lu J, Xiao P F, Bian X N, Liu H, Hu D X, Ling J, Li J, Zhai Z, Kong L J, Hu S Y
Department of Hematology and Oncology, Children's Hospital of Soochow University, Suzhou 215003, China.
Zhonghua Xue Ye Xue Za Zhi. 2018 Mar 14;39(3):231-235. doi: 10.3760/cma.j.issn.0253-2727.2018.03.011.
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). 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. 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). Low intensity of conditional regimen is efficient and safe which should be recommended for FA patients with HSCT.
评估低强度预处理方案对接受异基因造血干细胞移植（allo-HSCT）的范可尼贫血（FA）患儿的有效性和安全性。本研究纳入4例诊断为范可尼贫血的患者。1例接受了人类白细胞抗原（HLA）相合的同胞供者造血干细胞移植，2例接受了无关供者匹配（UD）的HSCT，1例接受了无关脐血移植。预处理方案为白消安联合小剂量环磷酰胺。4例均成功进行了allo-HSCT。中性粒细胞植入的中位时间为11（9-15）天，血小板（PLT）植入的中位时间为12（8-28）天。1例发生Ⅰ级急性移植物抗宿主病（aGVHD），1例发生出血性膀胱炎。无患者发生肝静脉闭塞病（VOD）。低强度预处理方案有效且安全，应推荐用于接受HSCT的FA患者。
