Treatment Efficacy and Lung Abnormalities of Amikacin Liposome Inhalation Suspension for Refractory Mycobacterium avium Complex Pulmonary Disease: A Two-Center Retrospective Cohort Study

Background : The guideline recommends the addition of amikacin liposome inhalation suspension (ALIS) to guideline-based therapy for patients with refractory Mycobacterium avium complex pulmonary disease (MAC-PD). The real-world data on the treatment efficacy and safety of ALIS remain insufficient. Research Question : What factors influence culture conversion during treatment with ALIS, and what are the best practices for managing ALIS-related adverse events, particularly ALIS-related lung abnormalities (ALIS-RLA)? Study Design and 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. On the basis of data obtained from medical records, adverse events were assessed in 76 patients, whereas treatment efficacy was evaluated in 49 patients. Results : The overall sputum culture conversion rate was 28.6%. However, among patients without cavities, it was significantly higher at 64.7%. Prognostic factors associated with culture conversion included a greater body mass index (19.5 kg/m ² vs. 17.2 kg/m ² , p=0.010), a lower rate of prior aminoglycoside administration (50.0% vs. 91.4%, p=0.003), a lower rate of cavity formation (21.4% vs. 82.9%, p<0.001), and a lower radiological severity, as calculated by the NICE scoring system (7.0 vs. 14.0, p=0.006). The most frequent adverse event was dysphonia, which occurred in 53.9% of the patients. Newly developed ALIS-RLA were observed in 82.1% of patients who underwent chest computed tomography (67 patients): 53 patients (79.1%) had multiple nodular patterns, 18 patients (26.9%) had the organizing pneumonia pattern, one patient (1.5%) had the diffuse alveolar pattern and another (1.5%) had the hypersensitivity pneumonitis pattern. Among patients with ALIS-RLA, 92.7% were asymptomatic, and 85.5% were able to continue ALIS. Interpretation : Introducing ALIS at an early stage is crucial for preventing the progression of MAC-PD. Although ALIS-RLA were observed in 82.1% of patients, discontinuation of ALIS appeared unnecessary in asymptomatic cases of ALIS-RLA.