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Real-world effectiveness and lung abnormalities associated with amikacin liposome inhalation suspension
Nozomi Tokita1, Masashi Ito2, Naohisa Urabe3
1Respiratory Disease Center, Fukujuji Hospital, Japan Anti-Tuberculosis Association, Tokyo, Japan; Department of Respiratory Medicine, Toho University Omori Medical Center, Tokyo, Japan.
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.
Insights
Amikacin liposome inhalation suspension (ALIS) shows a 28.6% culture conversion rate for refractory Mycobacterium avium complex pulmonary disease (MAC-PD). Most patients with ALIS-related lung abnormalities (ALIS-RLAs) were asymptomatic and continued treatment.
Area of Science:
- Pulmonology
- Infectious Diseases
- Pharmacology
Background:
- Guidelines recommend amikacin liposome inhalation suspension (ALIS) for refractory Mycobacterium avium complex pulmonary disease (MAC-PD).
- Limited data exist on factors influencing culture conversion and managing ALIS-related adverse events, including ALIS-related lung abnormalities (ALIS-RLAs).
Purpose of the Study:
- To evaluate the efficacy and safety of ALIS in patients with refractory MAC-PD.
- To identify factors associated with treatment outcomes and adverse events, particularly ALIS-RLAs.
Main Methods:
- A two-center retrospective cohort study of 87 patients with refractory MAC-PD receiving ALIS.
- Data on adverse events (n=76) and treatment outcomes (n=49) were collected from medical records.
- Refractory MAC-PD was defined as failure to achieve sputum culture conversion after ≥6 months of guideline-based therapy.
Main Results:
- Sputum culture conversion rate was 28.6%.
- Lower BMI (<18.5 kg/m²) and presence of cavities were associated with negative impact on culture conversion.
- Dysphonia (53.9%) was the most frequent adverse event. ALIS-RLAs occurred in 82.1% of patients, mostly asymptomatic (92.7%) and allowing continued ALIS treatment (85.5%).
Conclusions:
- Early initiation of ALIS before significant disease progression is crucial.
- Despite high incidence of ALIS-RLAs, treatment discontinuation was often unnecessary in asymptomatic patients.
- ALIS demonstrates potential in managing refractory MAC-PD, with careful monitoring for adverse events.
