Treatment of four cases of Fanconi anemia by allogeneic hematopoietic stem cell transplantation with low intensity conditional regimen
10.3760/cma.j.issn.0253-2727.2018.03.011
- VernacularTitle: 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
- Author:
Hui HOU
1
;
Yanhua YAO
;
Jun LU
;
Peifang XIAO
;
Xinni BIAN
;
Hu LIU
;
Yixi HU
;
Jing LING
;
Jie LI
;
Zong ZHAI
;
Lingjun KONG
;
Shaoyan HU
Author Information
1. Department of Hematology and Oncology, Children’s Hospital of Soochow University, Suzhou 215003, China
- Publication Type:Journal Article
- Keywords:
Fanconi anemia;
Hematopoietic stem cell transplantation;
Graft vs host disease;
Children
- From:
Chinese Journal of Hematology
2018;39(3):231-235
- CountryChina
- Language:Chinese
-
Abstract:
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.
