Imatinib Treatment in PDGFRA-Negative Childhood Hypereosinophilic Syndrome

Pediatr Blood Cancer. 2016 Jan;63(1):164-7. doi: 10.1002/pbc.25702. Epub 2015 Aug 8.

Abstract

We report a 4-year-old female who presented with severe hypereosinophilia (215.7 K/μl) and end-organ dysfunction. Extensive evaluation including whole exome sequencing was performed, revealing no causative mutation. Initial treatment with corticosteroids, leukapheresis, and hydroxyurea decreased her absolute eosinophil count (AEC), although it remained elevated. Despite the absence of a PDGFRA mutation, an imatinib trial resulted in normalization of her AEC. Imatinib was discontinued after sustained normal counts for 1 month. AECs have remained normal for more than 1 year off therapy. This provides support for consideration of imatinib in the treatment of hypereosinophilia even in the absence of a known tyrosine kinase mutation.

Keywords: PDGFRA; hypereosinophilia; imatinib; pediatric.

Publication types

  • Case Reports

MeSH terms

  • Child, Preschool
  • Female
  • Humans
  • Hypereosinophilic Syndrome / drug therapy*
  • Hypereosinophilic Syndrome / genetics
  • Imatinib Mesylate / therapeutic use*
  • Protein Kinase Inhibitors / therapeutic use
  • Receptor, Platelet-Derived Growth Factor alpha / analysis*
  • Sequence Analysis, DNA

Substances

  • Protein Kinase Inhibitors
  • Imatinib Mesylate
  • Receptor, Platelet-Derived Growth Factor alpha