BCR/ABL-Positive Chronic Myeloid Leukemia in Children: Current Treatment Approach

Curr Oncol Rep. 2024 Mar;26(3):250-257. doi: 10.1007/s11912-024-01502-z. Epub 2024 Feb 10.

Abstract

Purpose of review: The purpose of this review is to summarize the most updated treatment recommendations for pediatric CML, and to discuss current areas of investigation.

Recent findings: There is new phase 1 data to support the safety of the non-ATP competitive tyrosine kinase inhibitor (TKI) asciminib in the pediatric cohort. Ongoing studies are investigating the role of treatment-free remission in children. Chronic phase CML in children is managed with lifelong TKI therapy; however, evidence of deeper remissions sustained with second-generation TKIs may permit shorter treatment courses. Use of more specific TKIs may mitigate some of the side effects specific to the pediatric cohort. Children with advanced phase CML should achieve a complete hematologic remission with use of a second-generation TKI prior to transplant to achieve the best outcome.

Keywords: BCR-ABL1; CML; Chronic myeloid leukemia; Pediatric cancer; TKI; Tyrosine kinase inhibitor.

Publication types

  • Review

MeSH terms

  • Child
  • Fusion Proteins, bcr-abl / genetics
  • Humans
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive* / drug therapy
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive* / genetics
  • Leukemia, Myeloid*
  • Protein Kinase Inhibitors / therapeutic use

Substances

  • Fusion Proteins, bcr-abl
  • Protein Kinase Inhibitors