The Japanese Journal of Pediatric Hematology / Oncology
Online ISSN : 2189-5384
Print ISSN : 2187-011X
ISSN-L : 2187-011X
Present and future of clinical trial in pediatric acute myeloid leukemia: Clinical practice and research for children with cancer
Souichi Adachi
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2025 Volume 62 Issue 5 Pages 239-243

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Abstract

The Japan Children’s Cancer Group (JCCG) has conducted numerous clinical trials for pediatric acute myeloid leukemia (AML) of various subtypes; de novo AML (the most prevalent), AML associated with Down syndrome, acute promyelocytic leukemia, and relapsed AML. The AML-12 study, Japan’s first phase III randomized clinical trial for pediatric AML, demonstrated the prognostic value of measurable residual disease (MRD) assessed using multicolor flow cytometry. Currently, we are conducting the AML-20 study, which uses MRD status for patient stratification. However, pediatric AML outcomes have not significantly improved over the past two decades. Therefore, we aimed to develop novel therapies through close collaboration with the Japan Adult Leukemia Study Group and advance our understanding of disease biology through whole-genome sequencing analyses.

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© 2025 The Japanese Society of Pediatric Hematology / Oncology
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