Real-world use of liposomal amphotericin B: a large retrospective, observational, multicenter study

Background: Invasive fungal diseases (IFDs) cause significant morbidity and mortality, and liposomal amphotericin B (L-AmB) is a key treatment option for both IFDs and visceral leishmaniasis. Despite extensive clinical experience, real-world data on L-AmB use across diverse clinical settings remain limited. Methods: The AMPHO-SITA study was a multicenter, retrospective observational study conducted across 14 Italian centers (September 2020-October 2024). Adult patients receiving at least one dose of L-AmB were included. Baseline demographics, comorbidities, infection and L-AmB treatment characteristics, and outcomes were collected. IFDs were classified according to EORTC/MSGERC and FUNDICU definitions, and visceral leishmaniasis according to international guidelines. Primary descriptive endpoints were patient characteristics, infection characteristics, 30-day mortality, and 90-day mortality. Secondary analyses assessed factors associated with mortality using multivariable Cox regression models. Results: Among 389 patients (median age 61 years; 37.8% female), 41.3% had hematological malignancies and 52.4% required ICU admission. L-AmB was administered empirically in 53.0%, as targeted therapy in 47.0% of cases, and as combination therapy in 12.4%. Proven or probable IFDs were diagnosed in 52.2% of patients; visceral leishmaniasis was diagnosed and treated in 4.1%. Cumulative 30-day and 90-day mortality for IFD patients were 35.7% (95% confidence interval [CI] 28.5-42.9) and 61.4% (95% CI 51.8-69.6), respectively. NYHA score and SOFA score showed an independent association with both 30-day and 90-day mortality in multivariable models. Conclusions: L-AmB remains an important antifungal option in real-life, across diverse clinical scenarios and in heterogeneous patient populations with substantial comorbidity and clinical severity, while overall retaining a manageable safety profile.