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Real-World Efficacy and Safety of Liposomal Amphotericin B for Invasive Fungal Disease in Patients with Hematologic
Tianyu Lu1,2, Jianping Zhang3, Deyan Liu3
1Department of Hematology, Soochow Hopes Hematonosis Hospital, Suzhou, 215000, People's Republic of China.
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.
Insights
Domestic liposomal amphotericin B (L-AmB) shows good efficacy and safety in Chinese patients with hematologic malignancies and invasive fungal disease (IFD). Treatment success was achieved in 71% of patients, with a favorable benefit-risk balance, supporting L-AmB as a viable option.
Area of Science:
- Hematology
- Infectious Diseases
- Pharmacology
Background:
- Patients with hematologic malignancies face high risks of invasive fungal disease (IFD).
- Liposomal amphotericin B (L-AmB) is effective and well-tolerated, but real-world data on domestic L-AmB in China is limited.
- This study addresses the need for real-world evidence on domestic L-AmB in this vulnerable patient population.
Purpose of the Study:
- To evaluate the real-world efficacy and safety of domestic liposomal amphotericin B (L-AmB) in patients with hematologic malignancies and IFD in China.
- To assess the overall treatment success rate and safety profile, including adverse events (AEs).
- To determine the clinical benefit-risk balance of domestic L-AmB treatment.
Main Methods:
- Retrospective analysis of 100 patients with hematologic malignancies and IFD treated with domestic L-AmB in China.
- Efficacy assessed by overall treatment success; safety evaluated by adverse events (AEs).
- A composite outcome of treatment success without severe AEs (≥ Grade 3) was used to evaluate the benefit-risk balance.
Main Results:
- Overall treatment success rate was 71.0% (71/100) with a median treatment duration of 12 days.
- Domestic L-AmB demonstrated a favorable safety profile with mostly mild-to-moderate AEs; hypokalemia was most frequent (52.0%).
- 51% of patients achieved treatment success without severe AEs, indicating a favorable benefit-risk balance, particularly with treatment duration >14 days.
Conclusions:
- Domestic L-AmB is a clinically effective and well-tolerated treatment for invasive fungal disease in patients with hematologic malignancies.
- The findings support the use of domestic L-AmB in China, offering a valuable therapeutic option.
- Longer treatment durations may further enhance the benefit-risk profile of domestic L-AmB.
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