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Myelodysplastic syndromes (MDS) are a heterogeneous group of myeloid malignancies with a high risk of transformation into acute myeloid leukemia (AML) [1]. Pediatric MDS is extremely rare, with an annual incidence rate of 1–4 cases per million, accounting for less than 5% of hematologic malignancies in children [2].

骨髓增生异常综合征（MDS）是一组异质性髓系恶性肿瘤，具有转化为急性髓系白血病（AML）的高风险。小儿MDS极为罕见，年发病率为百万分之一至四例，占儿童血液系统恶性肿瘤的不到5%[2]。

Advanced MDS in children, including refractory anemia with excess blasts (RAEB) and RAEB in transformation (RAEB-T) [3], has a poor prognosis, with a 5-year overall survival (OS) of approximately 35–63% [4, 5].Currently, there is no standard treatment protocol for pediatric MDS, and limited data are available on children receiving heterogeneous regimens before transplants.

儿童晚期MDS，包括难治性贫血伴母细胞过多（RAEB）和转化中的RAEB（RAEB-T），预后不良，5年总生存率（OS）约为35-63%[4，5]。目前，儿科MDS没有标准的治疗方案，关于移植前接受异质方案的儿童的数据有限。

Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative treatment for pediatric MDS. Studies suggest that pre-transplant cytoreductive therapy, including demethylating treatment, low-dose chemotherapy, and AML-like chemotherapy, can improve the tumor burden before transplantation, reduce relapse, and improve long-term prognosis [6]..

异基因造血干细胞移植（HSCT）仍然是小儿MDS的唯一治疗方法。研究表明，移植前细胞减灭治疗，包括去甲基化治疗，低剂量化疗和AML样化疗，可以改善移植前的肿瘤负荷，减少复发，改善长期预后。。

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Fig. 1: Survival outcomes in 30 pediatric patients with advanced MDS.

图1:30例晚期MDS儿科患者的生存结果。

Data availability

数据可用性

Data are available on the basis of institutional review board-approved data request in alignment with applicable data protection regulations.

根据机构审查委员会批准的数据请求，根据适用的数据保护法规提供数据。

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Download referencesAcknowledgementsThis work was supported by the CAMS Innovation Fund for Medical Sciences grants (CIFMS) (2023-I2M-C&T-B-106 (WY), 2021-I2M-1-003 (WY), 2021-I2M-1-041 (YW), 2022-I2M-1-022 (XZ)), the National Natural Science Foundation of China (82270144 (XZ)), and the Health and Wellness Development Promotion grants-Huiyu Oncology Research grants (008 (WY)).Author informationAuthors and AffiliationsState 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, 300020, ChinaXingchen Wang, Chenmeng Liu, Yunlong Chen, Yang Wan, Wenbin An, Xiaolan Li, Lipeng Liu, Fang Liu, Li Zhang, Yao Zou, Xiaojuan Chen, Yumei Chen, Ye Guo, Xiaofan Zhu & Wenyu YangTianjin Institute of Health Science, Tianjin, 301600, ChinaXingchen Wang, Chenmeng Liu, Yunlong Chen, Yang Wan, Wenbin An, Xiaolan Li, Lipeng Liu, Fang Liu, Li Zhang, Yao Zou, Xiaojuan Chen, Yumei Chen, Ye Guo, Xiaofan Zhu & Wenyu YangAuthorsXingchen WangView author publicationsYou can also search for this author in.

下载参考文献致谢这项工作得到了CAMS医学科学创新基金（CIFMS）（2023-I2M-C＆T-B-106（WY），2021-I2M-1-003（WY），2021-I2M-1-041（YW），2022-I2M-1-022（XZ）），国家自然科学基金（82270144（XZ））和健康与健康发展促进基金会惠誉肿瘤学研究资助（008（WY））。作者信息作者和附属机构国家血液病临床研究中心实验血液学国家重点实验室，中国医学科学院和北京协和医学院血液与血液病研究所海河细胞生态实验室，天津，300020，中国王兴晨，刘晨梦，陈云龙，杨万，安文斌，李小兰，刘立鹏，刘芳，张丽，邹瑶，陈晓娟，陈玉梅，叶国，朱晓凡，杨宇文，天津卫生科学研究所，301600，王兴晨，刘晨梦，陈云龙，杨万，安文斌，李小兰，刘立鹏，刘芳，张丽，邹尧，陈晓娟，陈玉梅，叶果，朱晓凡和杨文玉作者星辰王维作者出版物您也可以在中搜索这位作者。

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PubMed Google ScholarContributionsXW was responsible for analyzing data, interpretation of results, visualization of data and results in figures and tables, and writing the manuscript. CL and YC was responsible for collecting data and provided conceptual input. YW, WA, XL, LL, FL, LZ, YZ, XC, YC and YG provided patient data.

PubMed Google ScholarContributionsXW负责分析数据，解释结果，可视化数据和图表中的结果，并撰写手稿。CL和YC负责收集数据并提供概念输入。YW，WA，XL，LL，FL，LZ，YZ，XC，YC和YG提供了患者数据。

XZ and WY designed the research and reviewed the manuscript. All authors agreed to submit the final version of the manuscript.Corresponding authorCorrespondence to.

XZ和WY设计了研究并审阅了手稿。所有作者都同意提交稿件的最终版本。对应作者对应。

Wenyu Yang.Ethics declarations

杨文宇。道德宣言

Competing interests

相互竞争的利益

The authors declare no competing interests.

作者声明没有利益冲突。

Ethics approval and consent to participate

道德批准和同意参与

All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki Declaration and its later amendments or comparable ethical standards. Exemption of informed consent approved by the Ethics Committee of Blood Diseases Hospital, Chinese Academy of Medical Sciences (IIT2021009-EC-1).

在涉及人类参与者的研究中进行的所有程序均符合机构和/或国家研究委员会的道德标准以及1964年《赫尔辛基宣言》及其后来的修正案或类似的道德标准。中国医学科学院血液病医院伦理委员会批准的知情同意豁免（IIT2021009-EC-1）。

Written informed consent was obtained from the guardian of the patient..

获得了患者监护人的书面知情同意书。。

Additional informationPublisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.Supplementary information41409_2024_2488_MOESM1_ESM.docxEfficacy and Prognostic Assessment of Chemotherapy-Bridged Transplantation in Pediatric Patients with Advanced Myelodysplastic SyndromesRights and permissionsReprints and permissionsAbout this articleCite this articleWang, X., Liu, C., Chen, Y.

Additional informationPublisher的注释Springer Nature在已发布的地图和机构隶属关系中的管辖权主张方面保持中立。补充信息41409\u 2024\u 2488\u MOESM1\u ESM.docx晚期骨髓增生异常综合征患儿化疗桥接移植的疗效和预后评估权利和许可证本文引用本文Wang，X.，Liu，C.，Chen，Y。

et al. Efficacy and prognostic assessment of chemotherapy-bridged transplantation in pediatric patients with advanced myelodysplastic syndromes..

化疗桥接移植治疗晚期骨髓增生异常综合征患儿的疗效和预后评估。。

Bone Marrow Transplant (2024). https://doi.org/10.1038/s41409-024-02488-9Download citationReceived: 19 September 2024Revised: 18 November 2024Accepted: 25 November 2024Published: 02 December 2024DOI: https://doi.org/10.1038/s41409-024-02488-9Share this articleAnyone you share the following link with will be able to read this content:Get shareable linkSorry, a shareable link is not currently available for this article.Copy to clipboard.

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