ArticleDrug design, development and therapy2026
Real-World Efficacy and Safety of Liposomal Amphotericin B for Invasive Fungal Disease in Patients with Hematologic Malignancies: A Multicenter Retrospective Study.
Article in Drug design, development and therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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11 authors.
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Abstract
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.
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