Abstract
Infant acute lymphoblastic leukemia (ALL), which develops in the first year of life, is a rare disease with approximately 20 cases per year in Japan. In particular, KMT2A (MLL) gene rearranged ALL (KMT2A-rALL) has a dismal prognosis, with a 5-year event-free survival rate of <50%. Moreover, acute and late severe toxicities from infants' intensive treatment remain an issue. Although outcomes of domestic and international clinical trials appear to improve gradually, the problem remains intractable. Therefore, introducing more appropriate risk stratification and less toxic and more effective novel treatment strategies is urgently required to improve the prognosis and long-term survival of infants with ALL. To achieve these goals, establishing new treatment strategies using novel agents through international collaborative studies is warranted in the future.
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