Liposomal amphotericin B (AmBisome) therapy in invasive fungal infections. Evaluation of United Kingdom compassionate use data.

BACKGROUND: Invasive fungal infections in the immunocompromised patient are associated with substantial mortality. The use of conventional amphotericin B, the main-stay of treatment, has often been limited by its adverse effects. The incorporation of amphotericin B into liposomes enables more drug to be given without an increase in adverse reactions. We examined the efficacy of AmBisome (Vestar Inc, San Diego, Calif), a small unilamellar liposomal formulation of amphotericin B, in the treatment of mycologically proven systemic fungal diseases.
METHODS: A retrospective analysis of the "Compassionate Use of AmBisome" in 58 patients who were treated in 34 centers throughout the United Kingdom between July 1990 and August 1992, before licensure of the drug.
RESULTS: Thirty patients had a definite or probable mycologic diagnosis, including 17 who had invasive aspergillosis, nine with Candida infections (three with mucosal disease only), three with zygomycosis, and one with cryptococcal meningitis. The overall response rate was 59% for patients with aspergillosis (80% for those who had had no prior therapy with amphotericin B) and 56% for those with candidosis. More than 40% of those in whom AmBisome was used as "salvage therapy" responded. A daily dose of up to 5 mg/kg was tolerated with minimal side effects.
CONCLUSIONS: AmBisome is efficacious in the treatment of invasive fungal infections and provides an alternative therapy for those who fail to respond or become intolerant to conventional amphotericin B therapy.