2023 Volume 60 Issue 2 Pages 130-138
Risk stratification is essential to the assignment of a patient to an appropriate therapy and prevention of excess toxicities, which has significantly contributed to an improvement in outcomes of pediatric acute myeloid leukemia (AML). Application of low-intensity chemotherapy to children with Down-syndrome-associated myeloid leukemia, whose normal and leukemic cells are both vulnerable to cytotoxic agents, is a good example. In children with de novo AML, cytogenetics and minimal/measurable residual disease evaluation are the most commonly used prognostic factors, which have been utilized to determine an indication of allogeneic hematopoietic stem cell transplantation. Additionally, with the recent advances in molecular biology and genetics, the identification of targetable lesions amenable to molecularly targeted therapy is becoming increasingly important. The introduction of all-trans-retinoic acid (ATRA) and/or arsenic trioxide to acute promyelocytic leukemia therapy is one of the advances successfully applied. With the expected near-future introduction of universal gene panel testing for hematological malignancies in Japan, further improvement of AML risk stratification is likely. However, the development of novel agents in Japan especially for children is lagging behind other countries, which should be urgently solved.
