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Updated: Nov 2, 2025

Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
How I treat pediatric acute myeloid leukemia
Jeffrey E Rubnitz1, Gertjan J L Kaspers2,3
1Department of Oncology, St Jude Children's Research Hospital, Memphis, TN.
Insights
Pediatric acute myeloid leukemia (AML) outcomes trail behind acute lymphoblastic leukemia (ALL) due to disease complexity and limited targeted therapies. Emerging immunotherapies and targeted agents offer new hope for improving treatment results in children with AML.
Area of Science:
- Pediatric Hematology Oncology
- Cancer Genomics
- Immunotherapy
Background:
- Pediatric acute myeloid leukemia (AML) survival rates lag behind acute lymphoblastic leukemia (ALL).
- Disease heterogeneity, limited targeted therapies, and slow immunotherapy development contribute to poor AML outcomes.
- Conventional chemotherapy intensification has reached its limits, necessitating novel therapeutic strategies.
Purpose of the Study:
- To highlight current challenges in pediatric AML treatment.
- To discuss emerging targeted therapies and immunotherapies for AML.
- To illustrate treatment controversies through case studies.
Main Methods:
- Review of current literature on pediatric AML treatment.
- Analysis of emerging therapeutic agents including venetoclax, CAR T-cell therapy, antibody therapy, and menin inhibitors.
- Presentation of four illustrative case studies of pediatric AML.
Main Results:
- Pediatric AML presents significant challenges due to its heterogeneous nature.
- New therapeutic avenues like BCL-2 inhibitors, CAR T-cell therapy, and menin inhibitors are showing promise.
- Case studies reveal ongoing controversies in optimal AML treatment strategies.
Conclusions:
- Novel targeted therapies and immunotherapies are crucial for advancing pediatric AML treatment.
- Further research and clinical trials are needed to optimize the use of these new agents.
- Personalized treatment approaches based on comprehensive genomic analysis are essential for improving outcomes.
Abstract:
Treatment outcomes for pediatric patients with acute myeloid leukemia (AML) have continued to lag behind outcomes reported for children with acute lymphoblastic leukemia (ALL), in part because of the heterogeneity of the disease, a paucity of targeted therapies, and the relatively slow development of immunotherapy compared with ALL. In addition, we have reached the limits of treatment intensity, and, even with outstanding supportive care, it is highly unlikely that further intensification of conventional chemotherapy alone will impact relapse rates. However, comprehensive genomic analyses and a more thorough characterization of the leukemic stem cell have provided insights that should lead to tailored and more effective therapies in the near future. In addition, new therapies are finally emerging, including the BCL-2 inhibitor venetoclax, CD33- and CD123-directed chimeric antigen receptor T-cell therapy, CD123-directed antibody therapy, and menin inhibitors. Here, we present 4 cases to illustrate some of the controversies regarding the optimal treatment of children with newly diagnosed or relapsed AML.
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