The guideline recommends the addition of amikacin liposome inhalation suspension (ALIS) for patients with refractory Mycobacterium avium complex pulmonary disease (MAC-PD). However, factors influencing culture conversion and managing ALIS-related adverse events, particularly ALIS-related lung abnormalities (ALIS-RLAs) remain insufficient.

This was a two-center retrospective cohort study involving 87 patients with refractory MAC-PD who received ALIS from August 2021 to September 2023. Refractory MAC-PD was defined as failure to achieve sputum culture conversion after six months or more of guideline-based therapy. On the basis of data obtained from medical records, adverse events were assessed in 76 patients, whereas treatment outcomes were evaluated in 49 patients.

The sputum culture conversion rate was 28.6 %. Prognostic factors associated with a negative impact included a body mass index below 18.5 kg/m² (adjusted odds ratio [aOR], 0.07, 95 % confidence interval [CI], 0.02–0.35) and the presence of cavities (aOR 0.02, 95 % CI 0.002–0.18). Patients achieved culture conversion had a shorter interval from MAC-PD treatment initiation to ALIS administration. The most frequent adverse event was dysphonia, which occurred in 53.9 %. ALIS-RLAs were observed in 82.1 %: 53 patients (79.1 %) had multiple nodular patterns, 18 patients (26.9 %) had organizing pneumonia patterns, one patient (1.5 %) had a diffuse alveolar pattern, and another (1.5 %) had a hypersensitivity pneumonitis pattern. Among patients with ALIS-RLAs, 92.7 % were asymptomatic, and 85.5 % were able to continue ALIS.

Initiating ALIS before significant progression is important. Although ALIS-RLAs were observed in 82.1 % of patients, discontinuation of ALIS appeared unnecessary in asymptomatic cases.