论著

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

  • 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
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  • 1.华中科技大学同济医学院(湖北武汉 430030)
    2.华中科技大学同济医学院附属武汉儿童医院血液肿瘤中心(湖北武汉 430030)
    3.武汉市第六医院/江汉大学附属医院肿瘤科(湖北武汉 430030)
    4.华中科技大学同济医学院附属武汉儿童医院儿童血液疾病研究室(湖北武汉 430030)
熊昊 电子信箱:xionghao@zgwhfe.com

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

  • WU Jinjun ,
  • XIONG Hao ,
  • ZENG Hui ,
  • CHEN Zhi ,
  • YANG Li ,
  • SUN Ming ,
  • WANG Zhuo ,
  • DU Yu ,
  • QI Shanshan ,
  • WANG Wei ,
  • ZHANG Lannan
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  • 1. Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430030, Hubei, China
    2. Department of Hematology and Oncology, Wuhan Children's Hospital,Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430016, Hubei, China
    3. Department of Radiotherapy and Oncology, Wuhan Sixth Hospital and Affiliated Hospital of Jianghan University, Wuhan 430015, Hubei, China
    4. Hematology Laboratory, Wuhan Children's Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430016, Hubei, China

Received date: 2025-08-19

  Accepted date: 2025-09-23

  Online published: 2026-01-05

摘要

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。

本文引用格式

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

Abstract

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.

参考文献

[1] Khoury JD, Solary E, Abla O, et al. The 5th edition of the world health organization classification of haematolymphoid tumours: myeloid and histiocytic/dendritic neoplasms[J]. Leukemia, 2022, 36(7): 1703-1719.
[2] Chung YJ, Bertoli R, Cao D, et al. Disruption of normal stem cell function and transmission of myelodysplastic syndrome by self-renewal of committed myeloid lineage cells[J]. Stem Cell Reports, 2025: 102571.
[3] Ramia DCM, Chen W. Myeloid sarcoma: an overview[J]. Semin Diagn Pathol, 2023, 40(3): 129-139.
[4] Goyal G, Bartley AC, Patnaik MM, et al. Clinical features and outcomes of extramedullary myeloid sarcoma in the United States: analysis using a national data set[J]. Blood Cancer J, 2017, 7(8): e592.
[5] Bianchi S, Capria S, Trisolini SM, et al. Myeloid sarcoma: diagnostic and treatment tools from a monocentric retrospective experience[J]. Acta Haematol, 2022, 145(1): 84-88.
[6] Shallis RM, Gale RP, Lazarus HM, et al. Myeloid sarcoma, chloroma, or extramedullary acute myeloid leukemia tumor: a tale of misnomers, controversy and the unresolved[J]. Blood Rev, 2021, 47: 100773.
[7] 中华医学会血液学分会干细胞应用学组, 中华医学会儿科学分会. 异基因造血干细胞移植治疗儿童急性淋巴细胞白血病中国专家共识(2022年版)[J]. 中华血液学杂志, 2022, 43(10): 793-801.
  Chinese expert consensus of the allogeneic hematopoietic stem cell transplantation for pediatric acute myeloid leukemia (not APL) (2022)[J]. Zhonghua Xueyexue Zazhi, 2022, 43(10): 793-801.
[8] Wang Y, Chang Y, Chen J, et al. Consensus on the monitoring, treatment, and prevention of leukaemia relapse after allogeneic haematopoietic stem cell transplantation in China: 2024 update[J]. Cancer Lett, 2024, 605: 217264.
[9] Gao L, Zhang Y, Wang S, et al. Effect of rhG-CSF combined with decitabine prophylaxis on relapse of patients with high-risk MRD-negative AML after HSCT: an open-label, multicenter, randomized controlled trial[J]. J Clin Oncol, 2020, 38(36): 4249-4259.
[10] 张宏, 于彤, 王岩, 等. 儿童髓系肉瘤的CT和MRI影像学表现分析[J]. 中国小儿血液与肿瘤杂志, 2022, 27(6): 383-389.
  Zhang H, Yu T, Wang Y, et al. Analysis of CT and MRI imaging manifestations of pediatric myeloid sarcoma[J]. Zhongguo Xiaoer Xueye Yu Zhongliu Zazhi, 2022, 27(6): 383-389.
[11] Duminuco A, Maugeri C, Parisi M, et al. Target therapy for extramedullary relapse of FLT3-ITD acute myeloid leukemia: emerging data from the field[J]. Cancers (Basel), 2022, 14(9): 2186.
[12] Magdy M, Abdel KN, Eldessouki I, et al. Myeloid sarcoma[J]. Oncol Res Treat, 2019, 42(4): 224-229.
[13] Zhao H, Dong Z, Wan D, et al. Clinical characteristics, treatment, and prognosis of 118 cases of myeloid sarcoma[J]. Sci Rep, 2022, 12(1): 6752.
[14] Zorn KE, Cunningham AM, Meyer AE, et al. Pediatric myeloid sarcoma, more than just a chloroma: a review of clinical presentations, significance, and biology[J]. Cancers (Basel), 2023, 15(5): 1443.
[15] Al Semari MA, Perrotta M, Russo C, et al. Orbital myeloid sarcoma (chloroma): Report of 2 cases and literature review[J]. Am J Ophthalmol Case Rep, 2020, 19: 100806.
[16] 潘虹宇, 吴南海, 吴敏媛, 等. 儿童髓系肉瘤临床特征及BCH-AML05方案治疗效果观察[J]. 人民军医, 2019, 62(6): 558-562.
  Pan HY, Wu NH, Wu MY, et al. Clinical characteristics of pediatric myeloid sarcoma and observation on the therapeutic effect of BCH-AML05 regimen[J]. Renmin Junyi, 2019, 62(6): 558-562.
[17] Lazzarotto D, Candoni A, Fili C, et al. Clinical outcome of myeloid sarcoma in adult patients and effect of allogeneic stem cell transplantation. Results from a multicenter survey[J]. Leuk Res, 2017, 53: 74-81.
[18] Ye F, Zhang H, Zhang W, et al. Clinical characteristics, pathology features and outcomes of pediatric myeloid sarcoma: a retrospective case series[J]. Front Pediatr, 2022, 10: 927894.
[19] 江亚军, 张春芳, 王红霞, 等. 原发性髓系肉瘤遗传学和分子学改变的临床意义[J]. 中国实验血液学杂志, 2024, 32(1): 27-32.
  Jiang YJ, Zhang CF, Wang HX, et al. Clinical significance of genetic and molecular alterations in primary myeloid sarcoma[J]. Zhongguo Shiyan Xueyexue Zazhi, 2024, 32(1): 27-32.
[20] Chiu AM, Yoon JG, Tirumani SH, et al. Myeloid sarcoma: a primer for radiologists[J]. J Comput Assist Tomogr, 2023, 47(3): 475-484.
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