A Simultaneous Assessment of Efficacy and Safety of Amikacin Liposome Inhalation Suspension for Refractory Mycobacterium avium Complex Lung Disease.

PURPOSE: Patients with refractory Mycobacterium avium complex lung disease (MACLD) represent a difficult-to-treat population with limited treatment options. In the CONVERT trial in refractory MACLD, amikacin liposome inhalation suspension (ALIS) plus multidrug therapy (MDT) resulted in higher culture conversion (CC) than MDT alone in the first 6 months of treatment (29% vs 9%). Adverse events (AEs) were reported in 98% and 91% of patients in the ALIS plus MDT and MDT alone arms, respectively, mostly of mild to moderate severity. A simultaneous assessment of efficacy and safety could be informative for clinical decision-making.

METHODS

A post hoc analysis of CONVERT was performed to compare time spent in a state of CC without severe AEs (CC-no severe AEs) between treatment with ALIS plus MDT and MDT alone. Time from randomization to month 6 per treatment arm was partitioned into 4 health states: (1) days spent with severe (≥grade 3) AEs prior to CC; (2) days spent without CC without severe AEs; (3) days spent with severe AEs after CC; and (4) days spent with CC-no severe AEs. The CC-no severe AEs, represented the only health state that provided both positive efficacy and safety treatment outcomes to patients. Mean differences in health-state durations across treatment arms with P values were calculated.

FINDINGS: Patients treated with ALIS plus MDT spent a mean of 24.3 more days in the health state of CC-no severe AEs than MDT alone (P < 0.001) over 6 months.

IMPLICATIONS: In this post hoc analysis of CONVERT, patients with refractory MACLD treated with ALIS plus MDT spent more time experiencing positive treatment outcomes compared with patients treated with MDT alone.