Treatment of acute myeloid leukemia in children: A practical perspective

Pediatr Blood Cancer. 2021 Jul;68(7):e28979. doi: 10.1002/pbc.28979. Epub 2021 Apr 12.


Pediatric acute myeloid leukemia (AML) is a heterogeneous disease that requires a multifaceted treatment approach. Although outcomes for low-risk AML have improved significantly over recent decades, high-risk AML continues to be associated with an adverse prognosis. Recent advances in molecular diagnostics, risk stratification, and supportive care have contributed to improvements in outcomes in pediatric AML. Targeted approaches, for example, the use of tyrosine kinase inhibitors to treat FLT3-ITD AML, offer promise and are currently undergoing clinical investigation in pediatric patients. New approaches to hematopoietic stem cell transplantation, including the use of haploidentical donors, are significantly expanding donor options for patients with high-risk AML. This review provides an overview of recent advances in the treatment of pediatric AML that are likely to have clinical impact and reshape the standard of care.

Keywords: HSCT; acute myeloid leukemia; childhood AML; pediatric AML.

Publication types

  • Review

MeSH terms

  • Child
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Leukemia, Myeloid, Acute* / drug therapy
  • Leukemia, Myeloid, Acute* / genetics
  • Mutation
  • Prognosis
  • Retrospective Studies
  • Transplantation, Homologous
  • fms-Like Tyrosine Kinase 3 / genetics


  • fms-Like Tyrosine Kinase 3