Very prolonged liposomal amphotericin B use leading to a lysosomal storage disease

Int J Antimicrob Agents. 2014 Jun;43(6):566-9. doi: 10.1016/j.ijantimicag.2014.02.015. Epub 2014 Apr 12.

Abstract

Amphotericin B is a powerful polyene antifungal drug used for treating systemic fungal infections and is usually administered for a short period. Side effects after prolonged use are unknown in humans. Here we report the case of a 28-year-old man suffering from chronic granulomatous disease (CGD), treated for invasive cerebral aspergillosis with liposomal amphotericin B (L-AmB) for a very long time (8 consecutive years). We describe the efficacy and safety of this treatment in the long term. Aspergillosis was kept under control as long as L-AmB therapy was maintained, but relapsed when the dose was reduced. No overt renal toxicity was noted. The patient gradually developed hepatosplenomegaly and pancytopenia. Abnormalities of bone marrow were similar to the sea-blue histiocyte syndrome. Liver biopsy showed images of nodular regenerative hyperplasia related to CGD as well as a histiocytic storage disease. We discuss the very prolonged use of L-AmB leading to the development of a lysosomal storage disease.

Keywords: Antifungal agents; Chronic granulomatous disease; Neuroaspergillosis; Sea-blue histiocyte syndrome.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Amphotericin B / adverse effects*
  • Amphotericin B / therapeutic use*
  • Antifungal Agents / adverse effects*
  • Antifungal Agents / therapeutic use*
  • Biopsy
  • Granulomatous Disease, Chronic / complications
  • Histocytochemistry
  • Humans
  • Liver / pathology
  • Lysosomal Storage Diseases / chemically induced*
  • Male
  • Neuroaspergillosis / drug therapy

Substances

  • Antifungal Agents
  • liposomal amphotericin B
  • Amphotericin B