Effective Use of ALK Inhibitors in EML4::ALK-Positive Lymphatic Malformations

Pediatr Blood Cancer. 2025 Feb;72(2):e31441. doi: 10.1002/pbc.31441. Epub 2024 Nov 11.

Abstract

Genetically targeted medications are emerging as important therapies for lymphatic malformations (LMs) unresponsive to sirolimus. We describe two patients with EML4::ALK-positive LMs, one with Gorham Stout disease and one with a large genitourinary (GU) LM, who were successfully treated with ALK inhibitors. This report adds ALK inhibitors to the growing toolbox of molecularly targeted therapies for LMs.

Keywords: hematology; molecular genetics; signal transduction therapeutics; vascular malformations.

Publication types

  • Case Reports

MeSH terms

  • Anaplastic Lymphoma Kinase / antagonists & inhibitors
  • Anaplastic Lymphoma Kinase / genetics
  • Crizotinib / therapeutic use
  • Female
  • Humans
  • Lymphatic Abnormalities* / drug therapy
  • Lymphatic Abnormalities* / genetics
  • Lymphatic Abnormalities* / pathology
  • Male
  • Oncogene Proteins, Fusion* / genetics
  • Protein Kinase Inhibitors / therapeutic use
  • Pyrazoles / therapeutic use

Substances

  • Oncogene Proteins, Fusion
  • Protein Kinase Inhibitors
  • Anaplastic Lymphoma Kinase
  • ALK protein, human
  • Crizotinib
  • EML4-ALK fusion protein, human
  • Pyrazoles