Abstract Rationale Guidelines recommend Amikacin liposome inhalation suspension (ALIS) for refractory Mycobacterium avium complex lung disease (MACLD) treatment, alongside other antibiotics. Efficacy of ALIS on microbiological endpoints and patient-reported outcomes (PROs) in the newly diagnosed MACLD population is unknown. Objectives ARISE aimed to validate Quality of Life–Bronchiectasis Respiratory Domain (QOL-B RD) and Patient-Reported Outcomes Measurement Information System Short Form v1.0-Fatigue 7a (PROMIS F SF-7a) in patients treated for new/recurrent MACLD. We present treatment outcome results, including microbiological endpoints, PROs, and safety. Methods Adults with noncavitary MACLD were randomized 1:1 to ALIS 590mg or empty liposome control (comparator), plus azithromycin 250mg and ethambutol 15mg/kg once daily for 6 months (M), then 1M off-treatment. Measurements and Main Results Of 99 patients, most had M. intracellulare (43.4%) and/or M. avium (32.3%) infections. Culture conversion with ALIS was achieved by 80.6% by M6 (comparator, 63.9%) and 78.8% by M7 (comparator, 47.1%; nominal P=0.0010). Among patients achieving culture conversion by M6, first negative culture defining conversion occurred at M1 for 74.3% with ALIS (comparator, 46.7%). Mean QOL-B RD score through M7 improved with ALIS vs plateauing with comparator after M3; both arms showed improved PROMIS F SF-7a without between-arm difference. Positive correlations between culture conversion and improved QOL-B RD score were observed with ALIS. No ALIS-related serious adverse events or deaths were reported. Conclusions More patients with newly diagnosed MACLD receiving 6M of ALIS alongside a macrolide-based regimen achieved culture conversion by M6 and M7 numerically more rapidly vs comparator. No new safety signals were identified.
Daley et al. (Fri,) studied this question.
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