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. 2025 May 13:18:2475-2487.
doi: 10.2147/IDR.S494985. eCollection 2025.

Efficacy and Safety of Amphotericin B Colloidal Dispersion for Patients with Invasive Fungal Disease and Febrile Neutropenia: A Registry-Based, Multicenter, Retrospective, Real-World Study

Li Zhang  1 , Qinglan Zhang  2 , Wei Ma  3 , Zhang-Bo Chu  4 , Li Xu  5 , Hongyun Xing  6 , Jishi Wang  7 , Jing Lin  1   8 , Zhengyin Liu  1
Affiliations

Efficacy and Safety of Amphotericin B Colloidal Dispersion for Patients with Invasive Fungal Disease and Febrile Neutropenia: A Registry-Based, Multicenter, Retrospective, Real-World Study

Li Zhang et al. Infect Drug Resist. .

Abstract

Purpose: This study aimed to explore the efficacy and safety of amphotericin B colloidal dispersion (ABCD) in treating patients with invasive fungal disease (IFD) or febrile neutropenia.

Patients and methods: This study retrospectively included patients diagnosed with IFD or febrile neutropenia who received ABCD treatment from 22 hospitals. The efficacy and safety were evaluated at the end of ABCD treatment. The characteristics of patients diagnosed with possible, probable and proven IFD according to the revised EORTC-MSG criteria were then further analyzed to conduct sensitivity analysis.

Results: A total of 503 patients were enrolled in this study. Of these patients, 391 received ABCD treatment for a minimum of seven days, and the overall efficacy of ABCD was determined to be 71.87% (281/391). 183 patients were diagnosed with possible, probable and proven IFD, the ABCD efficacy of whom was 67.76% (124/183). The efficacy of ABCD in patients with hematologic malignancies, AIDS and diabetes was 70.10% (211/301), 88.24% (30/34), and 83.33% (10/12), respectively. In terms of various fungal species, the efficacy of ABCD in patients with Aspergillus, Mucorales and Candida infections was 62.96% (34/54), 80.49% (33/41) and 66.67% (22/33), respectively. For patients in the targeted therapy, diagnostic-driven therapy, and empirical therapy groups, the efficacy of ABCD was 76.92% (60/78), 70.17% (207/295), and 77.78% (14/18), respectively. The most common adverse events (AEs) were infusion reactions (30.22%, 152/503) and hypokalemia (38.97%, 196/503), with the majority of these AEs classified as grade 1-2. This study was registered with ClinicalTrials.gov, NCT05116059.

Conclusion: ABCD has demonstrated satisfactory therapeutic efficacy and safety in the treatment of IFD or febrile neutropenia.

Keywords: amphotericin B colloidal dispersion; antifungal therapy; febrile neutropenia; invasive fungal disease.

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Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

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