Presentation of ichthyosis after substrate reduction therapy in Gaucher type 1

Am J Med Genet A. 2024 Dec;194(12):e63835. doi: 10.1002/ajmg.a.63835. Epub 2024 Aug 5.

Abstract

We describe a case in which a type 1 Gaucher patient developed ichthyosis weeks after starting substrate reduction therapy (SRT) with eliglustat. There are no reports of ichthyosis in the literature in enzyme replacement or SRT for Gaucher disease. Ichthyosis is seen with type 2 and 3 Gaucher disease, but not type 1. This raises the question: Why would a patient develop ichthyosis after starting SRT?

Keywords: Gaucher disease; case report; eliglustat; ichthyosis.

Publication types

  • Case Reports

MeSH terms

  • Enzyme Replacement Therapy
  • Female
  • Gaucher Disease* / complications
  • Gaucher Disease* / drug therapy
  • Gaucher Disease* / genetics
  • Glucosylceramidase / genetics
  • Glucosylceramidase / therapeutic use
  • Humans
  • Ichthyosis* / drug therapy
  • Ichthyosis* / pathology
  • Male
  • Pyrrolidines / therapeutic use

Substances

  • eliglustat
  • Pyrrolidines
  • Glucosylceramidase