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Targeted Therapy in Pediatric AML: An Evolving Landscape

LaQuita M Jones1,2, Katherine Tarlock3,4, Todd Cooper5,6

  • 1Department of Pediatrics, College of Medicine, University of Cincinnati, Cincinnati, OH, USA.

Paediatric Drugs
|August 22, 2021
PubMed

Insights

Pediatric acute myeloid leukemia (AML) outcomes have improved, but 30% of children remain uncured. This review summarizes targeted therapies for pediatric AML, leveraging genomic insights to improve treatment efficacy and reduce chemotherapy side effects.

Area of Science:

  • Pediatric Oncology
  • Hematology
  • Genomics

Background:

  • Outcomes for pediatric acute myeloid leukemia (AML) have improved due to intensive chemotherapy, hematopoietic stem cell transplant (HSCT), and supportive care.
  • Despite advancements, approximately 30% of pediatric AML cases remain uncured, highlighting the need for novel therapeutic strategies.
  • Current AML characterization employs advanced diagnostics like flow cytometry and comprehensive genomic sequencing.

Purpose of the Study:

  • To review current targeted therapy options for pediatric acute myeloid leukemia (AML).
  • To explore how genomic and molecular data inform the selection of targeted treatments.
  • To address the limitations of conventional chemotherapy and HSCT in pediatric AML.

Main Methods:

  • Literature review of current targeted therapy options in pediatric AML.
  • Analysis of diagnostic tools, including flow cytometry and genomic sequencing.
  • Synthesis of information on the genomic and molecular architecture guiding therapy selection.

Main Results:

  • Targeted therapies are increasingly utilized in pediatric AML, informed by molecular profiling.
  • Genomic insights are crucial for identifying specific targets and tailoring treatments.
  • This approach aims to improve cure rates and mitigate the toxicity associated with traditional therapies.

Conclusions:

  • Targeted therapies represent a promising avenue for improving outcomes in pediatric AML.
  • Understanding the molecular landscape of pediatric AML is essential for effective treatment selection.
  • Further research into targeted agents is critical to overcome treatment resistance and reduce long-term side effects.

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