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

J M Michot1, C Gubavu2, E Fourn2

  • 1Assistance Publique - Hôpitaux de Paris, Service de Médecine Interne et Immunologie Clinique, Hôpital Universitaire Bicêtre, 94275 Le Kremlin-Bicêtre Cedex, France; Université Paris Sud XI, 63 rue Gabriel Péri, 94276 Le Kremlin-Bicêtre Cedex, France.

International Journal of Antimicrobial Agents
|May 3, 2014
PubMed

Insights

This case study explores the long-term effects of liposomal amphotericin B (L-AmB) in a chronic granulomatous disease (CGD) patient. Prolonged L-AmB treatment controlled invasive aspergillosis but led to a rare lysosomal storage disease.

Area of Science:

  • Mycology
  • Pharmacology
  • Hematology

Background:

  • Amphotericin B is a critical antifungal agent for systemic infections.
  • Long-term side effects of liposomal amphotericin B (L-AmB) remain largely uncharacterized in humans.
  • Chronic granulomatous disease (CGD) predisposes patients to invasive fungal infections.

Observation:

  • A 28-year-old CGD patient received 8 years of continuous L-AmB for invasive cerebral aspergillosis.
  • Aspergillosis control correlated with L-AmB dosage, with relapse upon dose reduction.
  • The patient developed hepatosplenomegaly, pancytopenia, and bone marrow abnormalities resembling sea-blue histiocyte syndrome.

Findings:

  • Liposomal amphotericin B effectively managed invasive aspergillosis over an extended period.
  • No significant renal toxicity was observed during long-term L-AmB therapy.
  • Prolonged L-AmB administration was associated with the development of a lysosomal storage disease, characterized by nodular regenerative hyperplasia and histiocytic storage in the liver.

Implications:

  • This case highlights the potential for L-AmB to induce lysosomal storage disorders with very prolonged use.
  • It underscores the need for careful monitoring of hematological and hepatic parameters in patients on long-term antifungal therapy.
  • Further research is warranted to elucidate the mechanisms and long-term safety profile of extended L-AmB treatment.
Keywords:
Antifungal agentsChronic granulomatous diseaseNeuroaspergillosisSea-blue histiocyte syndrome

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