Real-world effectiveness and Lung Abnormalities Associated with Amikacin Liposome Inhalation Suspension.

Background: 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.

Methods: 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.

Results: 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.

Conclusion: 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.