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Published on: October 17, 2025
Prognostic factors and treatment of pediatric acute lymphoblastic leukemia
1Division of Hematology and Oncology, Department of Pediatrics, The Catholic University of Korea, College of Medicine, Seoul, Korea.
Insights
Pediatric acute lymphoblastic leukemia (ALL) survival has improved due to risk stratification and tailored treatments. Advances in targeted therapies and immunotherapy offer hope for high-risk and relapsed ALL patients.
Area of Science:
- Pediatric Oncology
- Hematology
- Clinical Trials
Background:
- Pediatric acute lymphoblastic leukemia (ALL) survival rates have significantly improved over recent decades.
- Accurate prognostic factor identification and risk-group stratification are key to enhanced outcomes in pediatric ALL.
- Cooperative clinical trials have guided treatment intensity and duration, leading to better results.
Purpose of the Study:
- To review the advancements in pediatric acute lymphoblastic leukemia (ALL) treatment and outcomes.
- To highlight the impact of risk stratification and targeted therapies on patient survival.
- To discuss current challenges and future directions in managing ALL, including relapsed cases and treatment toxicities.
Main Methods:
- Review of recent clinical trial data and therapeutic strategies for pediatric ALL.
- Analysis of prognostic factors influencing event-free survival (EFS) and overall survival.
- Evaluation of novel therapeutic approaches, including tyrosine kinase inhibitors and immunotherapy.
Main Results:
- Pediatric ALL treatment has evolved, with reduced reliance on cranial irradiation and improved central nervous system relapse rates.
- Philadelphia chromosome-positive (Ph+) ALL and infant ALL remain challenging subgroups with lower survival rates.
- Tyrosine kinase inhibitors have improved outcomes for Ph+ ALL, while infant ALL with MLL rearrangements still requires further therapeutic advancements.
- Immunotherapy targeting CD19 shows promise for relapsed and refractory ALL patients.
Conclusions:
- Continued progress in pediatric ALL management relies on refining risk stratification and optimizing treatment protocols.
- Addressing specific high-risk subgroups and relapsed/refractory disease through novel therapies is crucial.
- Future research should focus on minimizing long-term treatment toxicities while maintaining high survival rates.
Abstract:
The event-free survival (EFS) for pediatric acute lymphoblastic leukemia (ALL) has shown remarkable improvement in the past several decades. In Korea also, a recent study showed 10-year EFS of 78.5%. Much of the improved outcome for pediatric ALL stems from the accurate identification of prognostic factors, the designation of risk group based on these factors, and treatment of appropriate duration and intensity according to risk group, done within the setting of cooperative clinical trials. The schema of first-line therapy for ALL remains mostly unchanged, although many groups have now reported on the elimination of cranial irradiation in all patients with low rates of central nervous system relapse. Specific high risk subgroups, such as Philadelphia chromosome-positive (Ph+) ALL and infant ALL continue to have significantly lower survival than other ALL patients. The introduction of tyrosine kinase inhibitors into therapy has led to enhanced outcome for Ph+ ALL patients. Infant ALL patients, particularly those with MLL rearrangements, continue to have poor outcome, despite treatment intensification including allogeneic hematopoietic cell transplantation. Relapsed ALL is a leading cause of mortality in pediatric cancer. Recent advances in immunotherapy targeting the CD19 of the ALL blast have shown remarkable efficacy in some of these relapsed and refractory patients. With improved survival, much of the current focus is on decreasing the long-term toxicities of treatment.
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