Challenges and Efficacy of Amikacin Liposome Inhalation in Real-World Refractory Mycobacterium avium Complex Pulmonary Disease

Amikacin liposome inhalation suspension (ALIS) has emerged as a therapeutic option for patients with refractory <i>Mycobacterium avium</i> complex (MAC) pulmonary disease. This retrospective, observational study presents real-world data on the efficacy and safety of ALIS in 12 patients treated at our institution between July 2021 and November 2024. The cohort consisted predominantly of older patients (median age 72 years) with low BMI (median BMI 18.3). Radiographic patterns included nodular bronchiectatic (NB) and fibrocavitary (FC) types, with 75% of patients exhibiting cavitary lesions. Prior treatment regimens primarily involved azithromycin- or clarithromycin-based therapies, supplemented with sitafloxacin, with a median treatment duration of five years. ALIS therapy was discontinued in two cases due to adverse events, including fever and bronchospasm, while eight patients experienced hoarseness, and two reported hearing impairment or dizziness. Symptomatic improvement was observed in seven patients, and three of the nine patients who continued treatment for at least six months achieved culture conversion, all of whom exhibited the NB radiographic pattern. These findings highlight the challenges of managing refractory MAC pulmonary disease in a real-world setting and emphasize the importance of considering patient characteristics, including radiographic patterns and baseline health, when initiating ALIS therapy. Adverse events were frequent but manageable with appropriate monitoring and adjustments.