亲缘单倍体造血干细胞移植治疗KMT2A-r婴儿急性白血病临床疗效分析
Therapeutic effect of haploidentical hematopoietic stem cell transplantation for treatment of infant acute leukemia harboring KMT2A-r gene rearrangements
陈智 1王卓 1杨李 1卢文婕 1陶芳 1杜宇 1付强 1孙鸣 1祁闪闪 1熊昊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科,儿童血液医学研究室
- 折叠
摘要
目的 探讨亲缘单倍体造血干细胞移植(Haplo-HSCT)治疗KMT2A基因重排(KMT2A-r)婴儿白血病的疗效及安全性.方法 对2017年1月—2022年12月在本中心接受Haplo-HSCT的14例伴有KMT2A-r的婴儿白血病临床资料进行回顾性分析.结果 14例患儿中,急性淋巴细胞白血病(ALL)4例,急性髓系白血病(AML)9例,急性混合细胞白血病(MPAL)1例.患儿发病中位年龄为10.5(4-19)个月,接受移植中位年龄为16(7-26)个月.HLA配型均为亲缘单倍型相合,预处理均为Bu/Cy为基础的清髓方案,均接受经G-CSF动员的外周血造血干细胞移植.输入单个核细胞中位数为 19.95(7.6-31.5)×108/kg,CD34+细胞中位数为 12.01(1.46-20.45)× 106/kg.14 例患儿均获造血重建.1例因急性GVHD死亡,2例移植后复发最终死亡.存活11例患儿中位随访时间50(21-73)个月,预计3年总生存率(OS)为79％(11/14).结论 Haplo-HSCT治疗KMT2A-r婴儿急性白血病安全有效,仍需进一步减少移植相关并发症发生以改善患儿的长期存活率和生活质量.
Abstract
Objective To investigate the clinical efficacy and safety of haplotype hematopoietic stem cell transplantation(Haplo-HSCT)in the treatment of infantile leukemia with KMT2A-r gene rearrangement.Methods The clinical data of 14 infantile leukemia patients with KMT2A gene rearrangement who underwent Haplo-HSCT in our center from January 2017 to December 2022 were retrospectively analyzed.Results Among the 14 children,there were 4 cases of acute lymphoblastic leukemia(ALL),9 cases of acute myeloid leukemia(AML),and others with mixed phenotype acute leukemia(MPAL).The median age of onset of leukemia was 10.5(4-19)months,and the median age at receiving transplantation was 16(7-26months).The HLA matches were all related haplotype matched,and the conditioning regimen was all Bu/Cy-based myeloablative protocol,and all received peripheral blood hematopoietic stem cells mobilized by G-CSF.The median dosage of mononuclear cells was 19.95(7.6-31.5)× 108/kg,and the median number of CD34+cells was 12.01(1.46-20.45)× 106/kg.All 14 children reached hematopoietic reconstruction.One patient died of acute GVHD,and two patients died due to recurrence after transplantation.The median follow-up time of surviving children was 50 months(21～73months),and the 3-year overall survival rate(OS)was expected to reach 79％.Conclusions Haplo-HSCT is safe and effective in the treatment of KMT2A-r infantile leukemia,but transplantation-related complications need to be further reduced to improve the long-term survival rate and quality of life of infantile leukemia children.
关键词
亲缘单倍型造血干细胞移植/KMT2A基因重排/婴儿白血病Key words
Haploid hematopoietic stem cell transplantation/KMT2A-r/Infantile leukemia引用本文复制引用
基金项目
武汉市卫健委2020年度科研项目(WZ20Y04)
华中科技大学同济医学院附属武汉儿童医院腾飞计划()
武汉市卫健委2021年度科研项目(WZ21Z48)
武汉市卫生健康委临床医学项目-面上项目(WX21D60)
武汉市卫健委2020年度科研项目(WZ20D20)
武汉市应用基础前沿项目(2020020601012319)
出版年
2024
NETL
NSTL
万方数据