2025 Volume 62 Issue 5 Pages 239-243
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.