非亲缘供者造血干细胞移植治疗JMML的临床疗效  

The clinical efficacy of unrelated donor hematopoietic stem cell transplantation in the treatment of JMML

作　　者：彭智勇 宗飒[1] 汤浩然 裴艳茹 岳丹 李霞 何岳林 李春富 PENG Zhiyong;ZONG Sa;TANG Haoran;PEI Yanru;YUE Dan;LI Xia;HE Yuelin;LI Chunfu(Dongguan Taixin Hospital&Southern Chunfu Institute of Hematology,Dongguan 523128,China;Department of Pediatrics,Nanfang Hospital,Southern Medical University,Guangzhou 510515,China)

机构地区：[1]东莞台心医院&南方春富血液病研究院,广东省东莞市523000 [2]南方医科大学南方医院,广东省广州市515005

出　　处：《中国小儿血液与肿瘤杂志》2024年第5期317-321,326,共6页Journal of China Pediatric Blood and Cancer

基　　金：2023年国家自然科学基金面上项目(编号:82372312)。

摘　　要：目的评估非亲缘供者造血干细胞移植(URD-HSCT)治疗幼年粒单核细胞白血病(JMML)的临床疗效及安全性。方法回顾性分析2015年—2023年期间23例JMML移植患儿数据,中位移植年龄33(11-98)个月,PTPN11基因突变为主。供者选择HLA≥9/10位点相合的非亲缘供者。结果中位随访时间31(8-104)个月,输注CD34+中位数为4.8(1.3-12.5)×10^(6)/kg,23例均完全供者植入,粒细胞、血小板植入的中位时间分别为19天和13天,5年OS和LFS的统计预估值均为(91.1±6)%,无复发;Ⅱ度以上急性GVHD 7例(28%),Ⅲ-Ⅳ度仅1例;慢性GVHD 9例(39%),其中重度2例;移植相关死亡率8.9%。结论基于地西他滨的URD-HSCT方案治疗JMML临床疗效显著且安全,可作为JMML移植策略的优先推荐。Objective To evaluate the efficacy and safety of unrelated donor hematopoietic stem cell transplantation(URD-HSCT) in the treatment of Juvenile myelomonocytic leukemia(JMML).Methods Clinical data of 23 children with JMML who were treated with the same strategy in two transplantation centers from July 2015 to June 2023 were analyzed retrospectively.The median age at transplantation was 33(11-98) months.Of the 21 patients who could be evaluated,PTPN11 was the most common RAS pathway abnormal gene mutation.Unrelated donors with HLA high resolution matching ≥9/10 loci were selected.Results The median follow-up time was 31(8-104) months.All the 23 patients achieved complete donor engraftment.The median time of neutrophil and platelet engraftment was 19 days and 13 days,respectively.The 5-year overall survival(OS) and Leukemia-free survival(LFS) rates were both 91.1±6%.The incidence of grade Ⅱ or above aGVHD was 28%(7 cases),and grade Ⅲ~Ⅳ aGVHD occurred in 1 case.The incidence of chronic GVHD was 39%,with 2 cases of severe cGVHD.Transplant-related mortality was 8.9%.Conclusions Decitabine-based URD-HSCT has significant clinical efficacy and safety in the treatment of JMML,and can be recommended as a priority transplantation strategy for JMML.

关 键 词：非亲缘供者 造血干细胞移植 幼年粒单核细胞白血病 地西他滨 

分 类 号：R457.7[医药卫生—治疗学] R733.7[医药卫生—临床医学]