Efficacy and safety of amphotericin B colloidal dispersion in the treatment of mucormycosis: a real-world observation

BACKGROUND: Mucormycosis is a medical emergency associated with high morbidity and mortality, primarily affecting immunocompromised individuals. The treatment of mucormycosis remains challenging due to the limited effective antifungal options. This study aimed to assess the real-world efficacy and safety of long-term, low-dose amphotericin B colloidal dispersion (ABCD) in the treatment of mucormycosis. METHODS: This retrospective study at Huashan Hospital included mucormycosis patients treated with ABCD between August 1, 2021, and April 30, 2024. Efficacy and safety were assessed, and prognostic factors for 90-day mortality were identified using a Cox proportional hazards model. RESULTS: Thirty-eight patients were included. Pulmonary mucormycosis was the most common (60.5%) presentation followed by disseminated (23.7%), rhino-orbital (15.8%), and others. The predisposing factors included diabetes mellitus (55.3%), haematological malignancy (23.7%), COVID-19 infection (15.8%), and others. Rhizopus species (11/21, 52.4%) were the most common. ABCD was administered as primary therapy in 27 (71.1%) patients at a median dose of 2.3 mg/kg/day (interquartile range [IQR] 2.0–3.0) for 38 days (IQR 14–71). Seventeen patients (44.7%) received ABCD as salvage therapy, with a median dose of 2.5 mg/kg/day (IQR 1.9–3.1) over 31 days (IQR 22–68). Clinical response rates were 63.0% and 82.4% at the end of ABCD initial and salvage therapy, respectively. Adverse drug reactions occurred in 81.6% of patients, primarily hypokalemia (39.5%, 15/38). The 90-day mortality reached 15.8%. Glucocorticoid use was independently associated with reduced 90-day survival rate (hazard ratios 9.44, 95% confidence interval 1.85–48.32; p = 0.007). CONCLUSIONS: Long-term and low-dose use of ABCD had favorable safety and efficacy for both initial and salvage treatment of mucormycosis. Baseline glucocorticoid use was associated with higher 90-day mortality.