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.

Blood
|June 11, 2021
PubMed

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.

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