Aerosolized amikacin in patients with difficult-to-treat pulmonary nontuberculous mycobacteriosis.

Patients with pulmonary nontuberculous mycobacteriosis (pNTM) may have suboptimum response to conventional antimicrobial therapy. Aerosolized amikacin (aeAmk) was given to nine patients who had failed standard combination oral antimycobacterial drugs. A favorable toxicity profile, even in patients given aeAmk for an extended duration, median 75 ± 85 (range, 18-277) days and total cumulative dose 35,400 ± 30,568 (range, 7,600-95,400) mg, was encouraging, as was the clinical response and resolution of symptoms in 8 of 9 patients. The patient who failed therapy died due to complications arising from prior hematopoietic transplantation. The feasibility and efficacy of aeAmk in combination with oral anti-NTM drug(s) for treatment-refractory disease and, importantly, in primary therapy for pNTM requires validation randomized trials.