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Decitabine-containing conditioning improved outcomes for children with higher-risk myelodysplastic syndrome undergoing allogeneic hematopoietic stem cell transplantation

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Abstract

Myelodysplastic syndrome (MDS) is a rare clonal hematopoietic disorder in children. The risk stratification system and treatment strategy for adults are unfit for children. The role of hypomethylating agents (HMAs) in higher-risk childhood MDS has not been identified. This study aimed to investigate the outcomes of hematopoietic stem cell transplantation (HSCT) in children with higher-risk MDS at one single center. A retrospective study was conducted in children with higher-risk MDS undergoing HSCT between September 2019 and March 2023 at Blood Diseases Hospital CAMS. The clinical characteristics and transplantation information were reviewed and analyzed. A total of 27 patients were analyzed, including 11 with MDS with excess blasts (MDS-EB), 14 with MDS-EB in transformation (MDS-EBt) or acute myeloid leukemia with myelodysplasia-related changes (AML-MRC), and 2 with therapy-related MDS/AML (t-MDS/AML). Eight patients harbored monosomy 7. Before transplantation, induction therapy was administered to 25 patients, and 19 of them achieved bone marrow blasts <5% before HSCT. The stem cell source was unmanipulated-related bone marrow or peripheral blood stem cells for nineteen patients and unrelated cord blood for eight. All patients received decitabine-containing and Bu/Cy-based myeloablative conditioning; 26 patients achieved initial engraftment. The cumulative incidences of grade II–IV and grade III–IV acute graft-versus-host disease (GvHD) at 100 days were 65.4% and 42.3%, respectively. The incidence of cGvHD was 38.5%. The median follow-up was 26 (range 4–49) months after transplantation. By the end of follow-up, two patients died of complications and two died of disease progression. The probability of 3-year overall survival (OS) was 84.8% (95%CI, 71.1 to 98.5%). In summary, decitabine-containing myeloablative conditioning resulted in excellent outcomes for children with higher-risk MDS undergoing allogeneic HSCT.

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Acknowledgments

This study was supported by grants from the National Natural Science Foundation of China (82070201[Y.G.]) and the CAMS Innovation Fund for Medical Sciences (CIFMS) (2021-12M-1-041).

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Author notes

  1. Yuanyuan Ren, Fang Liu and Xia Chen contributed equally to this work.

Authors and Affiliations

  1. 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, 300020, China

    Yuanyuan Ren, Fang Liu, Xia Chen, Xiaoyan Zhang, Beibei Zhao, Yang Wan, Yang Lan, Xiaolan Li, Wenyu Yang, Xiaofan Zhu & Ye Guo

  2. Tianjin Institutes of Health Science, Tianjin, 301600, China

    Yuanyuan Ren, Fang Liu, Xia Chen, Xiaoyan Zhang, Beibei Zhao, Yang Wan, Yang Lan, Xiaolan Li, Wenyu Yang, Xiaofan Zhu & Ye Guo

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  1. Yuanyuan Ren
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  2. Fang Liu
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  3. Xia Chen
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Correspondence to Ye Guo.

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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.

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Ren, Y., Liu, F., Chen, X. et al. Decitabine-containing conditioning improved outcomes for children with higher-risk myelodysplastic syndrome undergoing allogeneic hematopoietic stem cell transplantation. Ann Hematol 103, 1345–1351 (2024). https://doi.org/10.1007/s00277-024-05628-9

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