Real-World Efficacy and Safety of Liposomal Amphotericin B for Invasive Fungal Disease in Patients with Hematologic Malignancies: A Multicenter Retrospective Study.

PURPOSE: Patients with hematologic malignancies are highly vulnerable to invasive fungal disease (IFD). Despite the broad-spectrum efficacy and lower toxicity of liposomal amphotericin B (L‑AmB), there is a lack of real‑world evidence on the recently approved domestic L‑AmB in China. This study aims to evaluate its real-world efficacy and safety in patients with hematologic malignancies.

PATIENTS AND

METHODS

This study retrospectively and consecutively included hematologic malignancies patients with IFD who received domestic L-AmB treatment in China between December 2024 and May 2025. Efficacy was evaluated by overall treatment success, and safety assessment was based on adverse events (AEs). A composite outcome of treatment success without severe AEs (≥ Grade 3) was used to evaluate the clinical benefit-risk balance.

RESULTS

A total of 100 eligible patients were included in this analysis, with a median duration of L-AmB treatment of 12 days (interquartile range [IQR], 7-19 days). The overall treatment success rate was 71.0% (71/100). Subgroup analysis demonstrated that longer treatment duration, acute lymphoblastic leukemia (ALL), and undefined or possible IFD categories were associated with a numerically higher treatment success rate. Furthermore, L-AmB exhibited a favorable safety profile, with mostly mild-to-moderate AEs. Hypokalemia was the most frequent (52.0%) but manageable, whereas nephrotoxicity, hepatotoxicity, and infusion-related reactions were mild. Notably, a total of 51 patients (51%) achieved treatment success without severe AEs, demonstrating an overall favorable benefit-risk balance. Dose-duration stratified composite outcome analysis showed that the longer treatment duration group (> 14 days) generally exhibited a superior benefit-risk balance (range 63.6%-84.6%).

CONCLUSION

Our findings support L-AmB as a clinically effective and well-tolerated therapeutic option for patients with hematologic malignancies complicated by IFD.