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Pediatric acute myeloid leukemia: towards high-quality cure of all patients
Gertjan J L Kaspers1, Christian M Zwaan
1Pediatric Oncology/Hematology, VU University Medical Center, De Boelelaan 1117 NL-1081 HV Amsterdam, The Netherlands. gjl.kaspers@vumc.nl
Insights
Childhood acute myeloid leukemia (AML) survival has improved, but intensive treatments cause side effects. Future therapies aim for better cure rates with targeted treatments for pediatric AML.
Area of Science:
- Pediatric Hematology/Oncology
- Cancer Genomics and Targeted Therapy
Background:
- Childhood acute myeloid leukemia (AML) prognosis has improved, with cure rates reaching approximately 60%.
- Current intensive chemotherapy regimens for pediatric AML lead to significant treatment-related mortality and long-term side effects.
Purpose of the Study:
- To review current and future classifications of pediatric AML.
- To discuss ongoing clinical trials and subgroup-directed treatment strategies.
- To explore novel therapeutic opportunities for improving cure rates in pediatric AML.
Main Methods:
- Review of current literature on pediatric AML classification and treatment.
- Discussion of risk-group stratification methods, including minimal residual disease monitoring, pharmacogenomics, and molecular abnormalities.
- Exploration of innovative therapies such as novel drug analogues, monoclonal antibodies, and tyrosine kinase inhibitors.
Main Results:
- Significant advancements in understanding pediatric AML biology.
- Development of new targeted drugs and therapeutic strategies.
- Potential for improved risk stratification to enable personalized treatment approaches.
Conclusions:
- Further refinement of pediatric AML treatment is needed to reduce toxicity.
- Precision medicine approaches, including targeted therapies, hold promise for improving outcomes.
- The goal is to achieve high-quality cure for nearly all children and adolescents with AML in the coming decades.
Abstract:
Prognosis of childhood acute myeloid leukemia (AML) has improved significantly over the past decades, from nearly no child surviving to a present probability of cure of approximately 60%. However, this can only be achieved using very intensive chemotherapy which results in relatively high rates of treatment related deaths and significant late effects. This review summarizes current and future classification of pediatric AML, ongoing phase III studies, and subgroup-directed treatment. In addition, the possibilities for more precise risk-group stratification which would allow more tailored and further refined subgroup-directed treatment are discussed. These include minimal residual disease monitoring, pharmacogenomics and the detection of AML-specific molecular abnormalities. Finally, we discuss the opportunities for innovative therapy in pediatric AML, such as the use of novel analogues, monoclonal antibody-mediated drugs, and receptor tyrosine kinase inhibitors. Given the enormous increase in our understanding of the underlying biology of AML, and the development of many new targeted drugs, it should be possible to achieve high-quality cure in nearly all children and adolescents with AML within the next few decades.
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