Skip to main content

ORIGINAL RESEARCH article

Front. Immunol.
Sec. Alloimmunity and Transplantation
Volume 15 - 2024 | doi: 10.3389/fimmu.2024.1426640

Decitabine-based treatment strategy improved the outcome of HSCT in JMML: a retrospective cohort study

Provisionally accepted
Zhiyong Peng Zhiyong Peng 1*Jingyu Gao Jingyu Gao 2Litao Huang Litao Huang 3Yuelin He Yuelin He 1Haoran Tang Haoran Tang 1Sa Zong Sa Zong 1Yanru Pei Yanru Pei 1Fuyu Pei Fuyu Pei 2Jing Ge Jing Ge 2Xuan Liu Xuan Liu 2Li Yue Li Yue 1Jun Zhou Jun Zhou 1Xia Li Xia Li 1Dan Yue Dan Yue 1Yun Chen Yun Chen 4Chen Chen Chen Chen 5Xuedong Wu Xuedong Wu 2Xiaoqin Feng Xiaoqin Feng 2Chunfu Li Chunfu Li 1
  • 1 Nanfang-Chunfu Children's Institute of Hematology and Oncology, Dongguan, China
  • 2 Department of Pediatrics, Nanfang Hospital, Southern Medical University, Guangzhou, China
  • 3 School of Forensic Medicine, Southern Medical University, Guangzhou, China
  • 4 Department of Hematology, Seventh Affiliated Hospital, Sun Yat-sen University, Shenzhen, China
  • 5 Other, Shanghai, China

The final, formatted version of the article will be published soon.

    Pre-HSCT disease control, suboptimal long-term prognosis, and a high recurrence incidence (RI) continue to pose significant challenges for hematopoietic stem cell transplantation (HSCT) in juvenile myelomonocytic leukemia (JMML) patients. This retrospective cohort study assessed the effectiveness of a decitabine (DAC)-based protocol in JMML patients undergoing HSCT.The pre-HSCT treatment includes initial and bridging treatment. The efficacy of DAC monotherapy versus DAC combined with chemotherapy (C-DAC) as initial treatment was compared, followed by DAC plus FLAG (fludarabine, cytarabine, and GCSF) as bridging treatment. The HSCT regimens were based on DAC, fludarabine, and busulfan. Post-HSCT, low-dose DAC was used as maintenance. The study endpoints focused on pretransplantation simplified clinical response and post-HSCT survival.There were 109 patients, including 45 receiving DAC monotherapy and 64 undergoing C-DAC treatment. 106 patients completed bridging treatment. All patients were administered planned HSCT regimens and post-HSCT treatment. The initial treatment resulted in 88.1% of patients achieving clinical remission without a significant difference between the DAC and C-DAC groups (p=0.769). Clinical remission rates significantly improved following bridging treatment (p=0.015). The 5-year overall survival, leukemia-free survival, and RI were 92.2%, 88.4%, and 8.0%, respectively. A poor clinical response to pre-HSCT treatment emerged as a risk factor for OS (hazard ratio: 9.8, 95% CI: 2.3-41.1, p=0.002). Implementing a DAC-based administration strategy throughout the pre-HSCT period, during HSCT regimens, and in post-HSCT maintenance significantly reduced relapse and improved survival in JMML patients. Both DAC monotherapy and the DAC plus FLAG protocol proved effective as pre-HSCT treatments.

    Keywords: Juvenile myelomonocytic leukemia (JMML)1, Decitabine2, FLAG chemotherapy3, hematopoietic stem cell transplantation (HSCT)4, Hypomethylating agents5

    Received: 01 May 2024; Accepted: 01 Aug 2024.

    Copyright: © 2024 Peng, Gao, Huang, He, Tang, Zong, Pei, Pei, Ge, Liu, Yue, Zhou, Li, Yue, Chen, Chen, Wu, Feng and Li. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

    * Correspondence: Zhiyong Peng, Nanfang-Chunfu Children's Institute of Hematology and Oncology, Dongguan, China

    Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.