| Summary: | Recent progress in risk-adapted treatment for childhood acute lymphoblastic leukemia has secured 5-year event-free survival rates of approximately 80% and 5-year survival rates approaching 90%. With improved systemic and intrathecal chemotherapy, it is now feasible to omit safely in all patients prophylactic cranial irradiation, which was once a standard treatment. As high-resolution, genome-wide analyses of leukemic and normal host cells continue to identify novel subtypes of lymphoblastic leukemia and provide new insights into leukemogenesis, we can look forward to the time when all cases of this disease will be classified according to specific genetic abnormalities, some of which will yield “druggable” targets for more effective and less toxic treatments. Meanwhile, it is sobering to consider that a significant fraction of leukemia survivors will develop serious health problems within 30 years of their initial diagnosis. This underlines the need to introduce early countermeasures to reduce late therapy-related effects. The ultimate challenge is to gain a clear understanding of the factors that give rise to childhood leukemia in the first place, and enable preventive strategies to be devised and implemented.
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