异基因造血干细胞移植联合化疗治疗儿童髓系肉瘤7例临床分析
收稿日期: 2025-08-19
录用日期: 2025-09-23
网络出版日期: 2026-01-05
基金资助
国家自然科学基金青年基金项目(8240003);湖北省自然科学基金青年项目(2024AFB410);湖北省自然科学基金创新发展联合基金项目(2024AFD435);武汉市自然科学基金探索计划市属医疗机构临床研究重点专项项目(2025020701020279)
Clinical analysis of allogeneic hematopoietic stem cell transplantation on pediatric myeloid sarcoma
Received date: 2025-08-19
Accepted date: 2025-09-23
Online published: 2026-01-05
目的 评估异基因造血干细胞移植(allo-HSCT)联合化疗治疗儿童髓系肉瘤(MS)的疗效。方法 回顾性分析于血液肿瘤中心确诊并治疗的7例MS患儿临床资料。结果 7例MS患儿中,男3例,女4例;诊断中位年龄5岁2个月;1例以“局部包块”就诊,6例在确诊急性髓细胞性白血病(AML)后发现存在髓外浸润;MS浸润部位包括头面部(4例)、纵隔(2例)、髂骨(1例)。7例患者均接受CCLG-AML-2019方案化疗并桥接allo-HSCT治疗。移植前均接受清髓预处理,其中5例采用后置环磷酰胺(PTCY),2例采用经典白消安联合环磷酰胺(BUCY)方案。7例患儿均回输G-CSF动员后采集的外周血造血干细胞,回输单个核细胞(MNC)中位数19.87×108/kg,CD34+细胞中位数12.87×106/kg。 中性粒细胞植入中位时间+15(13~15) d,血小板植入中位时间+12(11~17)d。随访截至2025年6月30日,中位随访时间1 008(371~1 814)d,5例存活,2例复发后死亡。无移植相关死亡发生,预计3年OS为71%。结论 早期诊断儿童MS仍存在挑战,规范化疗并尽早桥接allo-HSCT对部分患者能获得相对较好的疗效。
关键词: 髓系肉瘤; 急性髓细胞性白血病; 异基因造血干细胞移植; 儿童
吴进军 , 熊昊 , 曾辉 , 陈智 , 杨李 , 孙鸣 , 王卓 , 杜宇 , 祁闪闪 , 王伟 , 张兰男 . 异基因造血干细胞移植联合化疗治疗儿童髓系肉瘤7例临床分析[J]. 临床儿科杂志, 2026 , 44(1) : 44 -50 . DOI: 10.12372/jcp.2025.25e1007
Objective To explore the efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) combined with chemotherapy in children with myeloid sarcoma (MS). Methods A retrospective analysis was conducted on the clinical diagnosis, treatment and follow-up data of 7 children with MS who were diagnosed and treated at the hematology and oncology center from January 1, 2020 to December 31, 2023. Results There were 3 male and 4 female patients included in this study. The median age of diagnosis was 62 months (10 months - 14 years). Of the 7 patients, only 1 was initially presented with a "local mass," while the remaining 6 were diagnosed with acute myeloid leukemia (AML) and subsequently discovered to have extramedullary involvement, including 4 in the head and face, 2 in the mediastinum, and 1 in the ilium. All 7 patients received CCLG-AML-2019 protocol-based chemotherapy and bridging allo-HSCT. All children received a myeloablative pretreatment regimen prior to transplantation. Five of the 7 children were treated with post-infusion cyclophosphamide (PTCY), and 2 with busulfan combined with cyclophosphamide (BUCY). The median number of mononuclear cells infused was 19.87 (7.4-24.28)×108/kg, and the median number of CD34+ cells infused was 12.87 (7.1-14.7)×106/kg. The median time of neutrophil engraftment was +15(13-15) days, and the median time of platelet engraftment was +12(11-17) days. As of June 30, 2025, The median follow-up time was 1008 (371-1814) d. Five patients were alive, and 2 died. There was no death related to allo-HSCT. The estimated 3-year OS rate was 71%. Conclusion There are still challenges in the early diagnosis of MS in children, and the standard chemotherapy bridging allo-HSCT in the treatment of MS in children can obtain better efficacy.
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