中国儿童非唐氏综合征急性巨核细胞白血病患者：单中心真实世界分析
Pediatric non-Down's syndrome acute megakaryoblastic leukemia patients in China: A single center's real-world analysis.
作者信息
Zhang Aoli, Liu Lipeng, Zong Suyu, Chen Xiaoyan, Liu Chao, Chang Lixian, Chen Xiaojuan, Yang Wenyu, Guo Ye, Zhang Li, Zou Yao, Chen Yumei, Zhang Yingchi, Ruan Min, Zhu Xiaofan
机构信息
Department of Pediatric Hematology, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
Department of Hematology/Oncology, Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou, China.
出版信息
Front Oncol. 2022 Oct 4;12:940725. doi: 10.3389/fonc.2022.940725. eCollection 2022.
Non-Down's syndrome acute megakaryocytic leukemia (non-DS-AMKL) is a subtype of childhood acute myeloid leukemia (AML), whose prognosis, prognostic factors and treatment recommendations have not yet to be defined in children. We conducted a retrospective study with 65 newly diagnosed non-DS-AMKL children from August 2003 to June 2020 to investigate the clinical impact of factors and clinical outcome. Among all 65 patients, 47 of them were treated at our center who received three different regimens due to time point of admission (CAMS-another, CAMS-2009 and CAMS-2016 protocol), and the efficacy were compared. Patients with newly diagnosed non-DS-AMKL accounted for 7.4% of pediatric AML cases. The median age of the patients was 18 months at diagnosis, and over 90% of them were under three-years-old. The overall survival (OS) rates were 33.3% ± 1.7%, 66.7% ± 24.4% and 74.2% ± 4.0% for three groups (CAMS-another, CAMS-2009 and CAMS-2016 regimen), respectively. In CAMS-2016 group, the complete remission (CR) rate after induction was 67.7% (21/31), while the total CR rate after all phases of chemotherapy was 80.6% (25/31). The 2-year survival probability did not significantly improve in patients underwent HSCT when compared with non-HSCT group (75.0% ± 4.7% vs. 73.9% ± 4.6%, =0.680). Those who had a "dry tap" during BM aspiration at admission had significantly worse OS than those without "dry tap" (33.3% ± 8.6% vs. 84.0% ± 3.6%, 0.006). Moreover, the results also revealed that patients with CD34+ had significantly lower OS (50.0% ± 6.7% vs. 89.5% ± 3.5%, 0.021), whereas patients with CD36+ had significantly higher OS than those who were negative (85.0% ± 4.0% vs. 54.5% ± 6.6%, =0.048). In conclusion, intensive chemotherapy resulted in improved prognosis of non-DS-AMKL children and subclassification may base on "dry tap" and immunophenotypic. Although some progress has been made, outcomes of non-DS-AMKL children remain unsatisfactory, especially in HSCT group, when compared with other AML types.
非唐氏综合征急性巨核细胞白血病（non-DS-AMKL）是儿童急性髓系白血病（AML）的一种亚型，其预后、预后因素及治疗建议在儿童中尚未明确。我们对2003年8月至2020年6月新诊断的65例非DS-AMKL儿童进行了一项回顾性研究，以调查相关因素的临床影响及临床结局。在全部65例患者中，47例在本中心接受治疗，根据入院时间点接受了三种不同方案（CAMS-another、CAMS-2009和CAMS-2016方案），并比较了疗效。新诊断的非DS-AMKL患者占儿童AML病例的7.4%。患者诊断时的中位年龄为18个月，超过90%的患者年龄在3岁以下。三组（CAMS-another、CAMS-2009和CAMS-2016方案组）的总生存率（OS）分别为33.3%±1.7%、66.7%±24.4%和74.2%±4.0%。在CAMS-2016组中，诱导后完全缓解（CR）率为67.7%（21/31），而化疗各阶段后的总CR率为80.6%（25/31）。与非造血干细胞移植（HSCT）组相比，接受HSCT的患者2年生存概率无显著改善（75.0%±4.7%对73.9%±4.6%，P = 0.680）。入院时骨髓穿刺出现“干抽”的患者OS显著差于未出现“干抽”的患者（33.3%±8.6%对84.0%±3.6%，P = 0.006）。此外，结果还显示，CD34+患者的OS显著较低（50.0%±6.7%对89.5%±3.5%，P = 0.021），而CD36+患者的OS显著高于阴性患者（85.0%±4.0%对54.5%±6.6%，P = 0.048）。总之，强化化疗改善了非DS-AMKL儿童的预后，可根据“干抽”和免疫表型进行亚分类。尽管已取得一些进展，但与其他AML类型相比，非DS-AMKL儿童的结局仍不尽人意，尤其是在HSCT组。
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