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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

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.

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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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 (2022). https://doi.org/10.1038/s41409-022-01826-z

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