Amphotericin B Colloidal Dispersion Is Effective and Safe for the Treatment of Invasive Aspergillosis in Chinese Patients with Hematologic Disease: A Multicenter, Prospective, Observational, Real-World Study

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Purpose: Invasive aspergillosis (IA) causes significant morbidity and mortality in hematologic patients. This study aimed to evaluate the efficacy and safety profile of amphotericin B colloidal dispersion (ABCD) for IA in this high-risk population. Patients and Methods: This multicenter, prospective, observational study enrolled hematologic patients with IA. Eligible patients received ABCD for up to 28 days or until investigator-assessed clinical response or intolerance. The primary outcome was clinical response rate, and the secondary outcome was 28-day all-cause mortality following the last dose of ABCD. Safety was assessed based on the incidence and severity of adverse events (AEs). Results: From January 2022 to May 2024, a total of 65 patients were included. The median duration of ABCD treatment was 19 days (range, 1 to 28 days). Across all patients in the full analysis set (FAS), the clinical response rate was 64.62% (42/65). Whereas, in the per-protocol set (PPS), which comprised patients who completed at least 7 days of therapy, the response rate increased to 80.39% (41/51). Notably, only two patients died (3.08%) within 28 days following the last dose of ABCD. Furthermore, exploratory subgroup analysis revealed higher response rates in patients with possible IA, Eastern Cooperative Oncology Group performance status score (ECOG) score of 0– 2, baseline body temperature below 38°C, and acute lymphoblastic leukemia. Treatment-related AEs were predominantly grade 1– 2, demonstrating a favorable safety profile. The absence of grade 3/4 elevated creatinine events indicated the low nephrotoxicity of ABCD. Univariate and multivariate analyses of clinical response identified longer duration of ABCD treatment as the only independent predictor of clinical response. Nevertheless, the study predominantly included patients with possible IA and employed an observational single-arm design, and the findings should therefore be interpreted cautiously and may not be directly generalizable to populations with microbiologically confirmed IA. Conclusion: Our findings support ABCD as an effective and well-tolerated treatment in hematologic patients with IA, thus providing a reliable therapeutic option for managing IA in this high-risk population. Keywords: amphotericin B colloidal dispersion, invasive aspergillosis, efficacy, safety

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