Aerosolized amphotericin B inhalations as prophylaxis of invasive aspergillus infections during prolonged neutropenia: Results of a prospective randomized multicenter trial

We performed a prospective, randomized, multicenter trial to evaluate the e ffectiveness of prophylactic inhalations with aerosolized amphotericin B (a eroAmB) to reduce the incidence of invasive aspergillus (IA) infections in patients after chemotherapy or autologous bone marrow transplantation and a n expected duration of neutropenia of at least 10 days. From March 1993 unt il April 1996, 382 patients with leukemias, relapsed high-grade non-Hodgkin lymphomas, or solid tumors were randomized with a 13:10 ratio to receive e ither prophylactic aeroAmB inhalations at a dose of 10 mg twice daily or no inhalation prophylaxis in an unblinded fashion. The incidence of proven, p robable, or possible IA infections was 10 of 227 (4%) in patients who recei ved prophylactic aeroAmB. This did not differ significantly from the 11 of 155 (7%) incidence in patients who received no inhalation prophylaxis (P=.3 7). Moreover, no differences in the overall mortality (13% v 10%; P =.37) o r in the infection-related mortality (8% v 7%; P =.79) were found. In contr ast to other nonrandomized trials, we observed no benefit from prophylactic aeroAmB inhalations, but the overall incidence of IA infections was low. ( C) 1999 by The American Society of Hematology.