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Allogeneic hematopoietic cell transplantation in patients with juvenile myelomonocytic leukemia in Korea: a report of the Korean Pediatric Hematology-Oncology Group

Bone Marrow Transplantation volume 58, pages 20–29 (2023)Cite this article

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Abstract

Juvenile myelomonocytic leukemia (JMML) is a life-threatening myeloproliferative neoplasm. This multicenter study evaluated the characteristics, outcomes, and prognostic factors of allogeneic hematopoietic cell transplantation (HCT) in recipients with JMML who were diagnosed between 2000 and 2019 in Korea. Sixty-eight patients were retrospectively enrolled—28 patients (41.2%) received HCT during 2000–2010 and 40 patients (58.8%) during 2011–2020. The proportion of familial mismatched donors increased from 3.6 to 37.5%. The most common conditioning therapy was changed from Busulfan/Cyclophosphamide-based to Busulfan/Fludarabine-based therapy. The 5-year probabilities of event-free survival (EFS) and overall survival (OS) were 52.6% and 62.3%, respectively. The 5-year incidence of transplant-related mortality was 30.1%. Multivariate analysis revealed that the proportion of hemoglobin F ≥ 40%, abnormal cytogenetics, and matched sibling donors were independent risk factors for a higher relapse rate. Patients whose donor chimerism was below 99% had a significantly higher relapse rate. Better OS and lower treatment-related mortality were observed in patients with chronic graft-versus-host disease (GVHD), whereas grade III or IV acute GVHD was associated with worse EFS. In conclusion, the number of transplant increased along with the increase in alternative donor transplants, nevertheless, similar results were maintained. Alternative donor transplantation should be encouraged.

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Fig. 1: Cumulative incidence (CI) of relapse.
Fig. 2: Cumulative incidence (CI) of relapse.
Fig. 3: Acute graft-versus-host disease (aGVHD) and outcomes.
Fig. 4: Chronic graft-versus-host disease (cGVHD) and outcomes.

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Data availability

All the collected medical information was stored in the Internet-based Clinical Research and Trial Management System of the Korea Disease Control and Prevention Agency. The data that support the findings of this study are available from the corresponding author upon reasonable request.

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Acknowledgements

This study was supported by a grant from the Korea Childhood Leukemia Foundation and the Korean Pediatric Hematology-Oncology Group (2020). We also thank the Korea Disease Control and Prevention Agency for providing the Internet-based Clinical Research and Trial Management System for storing medical information online.

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Author notes

  1. These authors contributed equally: Eun Sang Yi, Seong Koo Kim.

Authors and Affiliations

  1. Department of Pediatrics, Korea University Guro Hospital, Korea University College of Medicine, Seoul, Korea

    Eun Sang Yi

  2. Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea

    Eun Sang Yi, Hee Young Ju & Keon Hee Yoo

  3. Department of Pediatrics, Seoul St. Mary’s Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea

    Seong Koo Kim, Jae Wook Lee & Bin Cho

  4. Department of Pediatrics, Seoul National University College of Medicine, Seoul National University Cancer Research Institute, Wide River Institute of Immunology, Seoul National University Children’s Hospital, Seoul, Korea

    Bo Kyung Kim & Hyoung Jin Kang

  5. Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun, Korea

    Hee Jo Baek & Hoon Kook

  6. Department of Pediatrics, Pusan National University School of Medicine, Pusan National University Children’s Hospital, Yangsan, Korea

    Eu Jeen Yang & Young Tak Lim

  7. Department of Pediatrics, Yonsei University College of Medicine, Severance Hospital, Seoul, Korea

    Won Kee Ahn & Seung Min Hahn

  8. Department of Pediatrics, Ulsan University Hospital, University of Ulsan College of Medicine, Ulsan, Korea

    Sang Kyu Park

  9. Department of Pediatrics, Ewha Womans University College of Medicine, Ewha Womans University Seoul Hospital, Seoul, Korea

    Eun Sun Yoo

  10. Department of Health Science and Technology, SAIHST, Sungkyunkwan University, Seoul, Korea

    Keon Hee Yoo

  11. Cell & Gene Therapy Institute, Samsung Medical Center, Seoul, Korea

    Keon Hee Yoo

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  1. Eun Sang Yi
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Contributions

BC, HJK, HK, YTL, and KHY designed this study. ESY, SKK, HYJ, JWL, BKK, HJB, EJY, WKA, SMH, SKP, and ESY supported data collection. ESY analyzed the data. ESY, SKK, and KHY drafted the original paper. All authors have reviewed and edited the paper. KHY supervised the study.

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Correspondence to Keon Hee Yoo.

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Yi, E.S., Kim, S.K., Ju, H.Y. et al. Allogeneic hematopoietic cell transplantation in patients with juvenile myelomonocytic leukemia in Korea: a report of the Korean Pediatric Hematology-Oncology Group. Bone Marrow Transplant 58, 20–29 (2023). https://doi.org/10.1038/s41409-022-01826-z

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