单倍体造血干细胞移植治疗儿童骨髓增生异常综合征的临床分析
Clinical analysis of haploid hematopoietic stem cell transplantation in pediatric patients with myelodysplastic syndrome
汪洁 1熊昊 1陈智 1杨李 1陶芳 1孙鸣 1卢文婕 1祁闪闪1
作者信息
- 1. 430016 武汉,华中科技大学同济医学院附属武汉儿童医院血液肿瘤科
- 折叠
摘要
目的 探讨儿童骨髓增生异常综合征(MDS)接受单倍体异基因造血干细胞移植(Haplo-HSCT)治疗的临床疗效,并分析预后相关因素.方法 回顾性分析2018年6月—2022年2月在武汉儿童医院血液肿瘤科接受Haplo-HSCT治疗的13例MDS患儿临床资料,并分析影响预后的危险因素.结果 13例儿童MDS患者中,男9例,女4例,中位年龄为9(1-13)岁,病史中位时间3.2年(2个月-10年),7例诊断难治性贫血伴原始细胞增多(RAEB),3例诊断难治性血细胞减少症(RCC),2例诊断RAEB向白血病转化或转化中RAEB-t,1例为难治性贫血伴铁粒幼细胞增多(RARS).患儿均接受亲缘供者(父亲、母亲、姐姐或弟弟),HLA配型为5-8/10位点相合,G-CSF动员的外周血干细胞,回输单个核细胞中位数22.17(16-32)×108/Kg,CD34+细胞数中位数11.5(7-14)x106/Kg.13例患儿均采用后置环磷酰胺方案预防急性移植物抗宿主病.粒细胞植入中位时间+14(11-18)d,血小板植入中位时间+16(10-19)d.中位随访时间为955(210-1700)d.13例患儿中1例因脓毒性休克死亡,3例合并肺部慢性排异,1例合并卵巢早衰.预期4年总体生存率为92.3％,非复发死亡率为7.7％.结论 接受Haplo-HSCT治疗儿童MDS疗效显著,移植相关并发症仍是患儿长期生存质量的严峻挑战.
Abstract
Objective To investigate the clinical efficacy of haploid hematopoietic stem cell transplantation(Haplo-HSCT)in pediatric patients with myelodysplastic syndrome(MDS),and analyze the prognostic factors.Methods The data of 13 pediatric patients with MDS who received haplo-HSCT in the Department of Hematology and Oncology of Wuhan Children's Hospital from June 2018 to February 2022 were retrospectively analyzed,prognostic risk factors were also analyzed.Results In 13 pediatric patients with MDS,there were 9 males and 4 females,with a median age of 9 years(range 1 year to 13 years old),and the average disease history was 3.2years(range from 2 months to 10 years).Seven patients were diagnosed with refractory anemia accompanied by erythrocytosis(RAEB),three patients were diagnosed with refractory cytopenia(RCC),two patients were diagnosed with RAEB-t in the transformation of RAEB-to leukemia,and one patient were diagnosed with refractory anemia accompanied by sideroblastosis(RARS).All the patients received peripheral blood stem cells mobilized by G-CSF from related donors(father,mother,sister or brother)with HLA matching of 5-8/10 sites,and the median mononuclear cells of 22.17(16-32)x 108/Kg were infused,median CD34+cells was 11.5(7-14)x 106/Kg post cyclophosphamide regimen was applied to prevent acute graft-versus-host disease,The median granulocyte engraftment was+14(11-18)days,and the median platelet engraftment was+16(10-19)days.The median follow-up time was 955(210-1700)days;1 of the 13 patients died of septic shock,3 had chronic pulmonary rejection,and 1had premature ovarian failure.The expected 4-year overall survival was 92.3％,non recurrent mortality rate was 7.7％.Conclusions Haploid hematopoietic stem cell transplantation in the treatment of pediatric patients with MDS can achieve an ideal long-term survival expectation,transplantation-related complications are still a serious challenge to the quality of life for pediatric patients.
关键词
骨髓增生异常综合征/单倍体造血干细胞移植/儿童Key words
Myelodysplastic syndrome(MDS)/Haploid hematopoietic stem cell transplantation(Haplo-HSCT)/Pediatric patients引用本文复制引用
基金项目
武汉市卫生健康委临床医学科研项目-青年项目(WZ20Y04)
武汉市卫生健康委-临床医学科研项目-面上一般项目(WX20D20)
武汉市卫生健康委-临床医学科研项目-面上一般项目(WX21D60)
湖北省自然科学基金-青年基金(2020CFB364)
武汉市科技局-应用基础研究计划(2020020601012319)
出版年
2024
NETL
NSTL
万方数据