A Novel Drug Formulation for Nontuberculous Mycobacterial Pneumonia

摘要

Liposomal amikacin for inhalation resulted in higher sputum conversion rates and increased exercise tolerance. Treatment for patients with pulmonary nontuberculous mycobacterial (PNTM) disease is often unsatisfactory. The addition of parenteral aminoglycosides, such as amikacin, to oral therapy is limited because of systemic toxicity. Liposomal amikacin for inhalation (LAI) is a novel formulation that allows for direct delivery of the drug to infected pulmonary macrophages with reduced toxicity. To examine the efficacy and safety of LAI for PNTM, investigators conducted a multicenter, randomized, double-blind, placebo-controlled, phase II, trial involving 89 PNTM patients with positive sputum cultures who had received multidrug therapy for >6 months prior to screening. Patients received a multidrug regimen plus either LAI or placebo by nebulizer once daily for 84 days. An additional 84 days of open-label therapy was given to consenting subjects. Among all patients, 64% had predominantly Mycobacterium avium complex infection, 36% had predominantly Mycobacterium abscessus infection, and 19% had cystic fibrosis.