Inhaled Amphotericin B as Aspergillosis Prophylaxis in Hematologic Disease: An Update

This review summarizes the literature on inhaled amphotericin B for invasive aspergillosis prophylaxis in patients with neutropenia secondary to hematologic malignancy treatment or stem cell transplant. Six trials, 2 randomized controlled and 4 with historical controls, were identified. Three inhaled amphotericin B deoxycholate trials found a reduced invasive aspergillosis incidence, 1 reaching statistical significance. Three inhaled liposomal amphotericin B trials demonstrated similar reductions with 2 finding statistical significance. Relative risk reductions for invasive aspergillosis were routinely 40-60%. Both formulations were without reported systemic or severe adverse effects. The most common adverse events were cough, bad taste, and nausea. Discontinuation rates ranged from 0-45%. The only randomized, placebo-controlled trial utilized inhaled liposomal amphotericin B reported a nearly 60% relative risk reduction. Inhaled liposomal amphotericin B 12.5 mg twice weekly is an alternative for invasive aspergillosis prophylaxis in high risk neutropenic patients with hematologic malignancies and stem cell transplant recipients when recommended azole agents are contraindicated or should not be used.