Evaluation of the Efficacy, Safetyand Economy of Different Amphotericin B Formulationsin Invasive Fungal Disease: A Retrospective Cohort Study postprint

Abstract: Background　Fungal infections have become an increasingly severe public health challenge worldwide. Amphotericin B is widely used in antifungal therapy and is available in four formulations: Amphotericin B Deoxycholate, Liposomal Amphotericin B, Amphotericin B Colloidal Dispersion, and Amphotericin B Lipid Complex. Despite having the same active ingredient, these different formulations may exhibit variations in efficacy, safety and cost-effectiveness. Objective　This study aims to investigate the differences in efficacy, safety, and cost-effectiveness among different formulations of amphotericin B in patients with invasive fungal disease (IFD). Methods　This retrospective observational study enrolled 71 IFD patients treated with amphotericin B at the First Hospital of Hebei Medical University from June 2023 to March 2025, who were divided into the AMB-D group (n=20), L-AMB group (n=20) and ABCD group (n=31) by the formulation type. The primary outcome was the effective remission rate of IFD, which was analyzed with Kaplan-Meier survival curves and the Log-rank test along with the impact of adverse events on survival. The incidences of renal injury, hepatic injury, hypokalemia and thrombocytopenia were compared to assess medication safety, and pharmacoeconomics was evaluated by analyzing total cost, daily average cost and cost-effectiveness. Results　A total of 71 patients were included in the study, with a median age of 64.0 (54.0, 71.0) years. Among them, 50 were males (70.4%) and 21 were females (29.6%). The complete response rate, partial response rate, and non-response rate of the included patients were 26.8% (19/71), 38.0% (27/71), and 35.2% (25/71), respectively. There were no statistically significant differences in the overall response rate and non-response rate among the AMB-D group, L-AMB group, and ABCD group (all P>0.05). Stratified analysis showed that the overall response rates differed significantly among the three groups in patients aged 61-83 years, females, and those with a tentative diagnosis (all P<0.05). In addition, the overall response rate and complete response rate differed significantly among the three groups when the treatment duration was less than 3 days (all P<0.05); the overall response rate differed significantly among the three groups when the treatment duration exceeded 7 days (P<0.05). Adverse events occurred in 49 of the 71 patients (69.0%), with the most common events being renal injury (31cases, 43.7%), hepatic injury (18 cases, 25.4%), hypokalemia (9 cases, 12.7%), and thrombocytopenia (17 cases, 23.9%). Survival analysis revealed no statistically significant differences in survival time among the three groups for patients who developed renal injury, hepatic injury, hypokalemia, or thrombocytopenia (all P>0.05). There were statistically significant differences in the total cost and daily cost among the three groups (all P<0.01), with the L-AMB group showing the highest cost-effectiveness ratio. Conclusion　Amphotericin B formulations exhibit marked variations in efficacy and economy profiles, necessitating individualized selection guided by specific clinical characteristics. Rigorous monitoring of renal function remains imperative throughout the therapeutic course.