Amikacin Liposome Inhalation Suspension to Treat M Abscessus Pulmonary Disease

What outcomes are associated with compassionate use of amikacin liposome inhalation suspension for patients with Mycobacterium abscessus pulmonary disease?

In patients with Mycobacterium abscessus pulmonary disease, treatment with amikacin liposome inhalation suspension (ALIS) is associated with manageable respiratory adverse events (AEs), with outcomes consistent with those from guideline-based treatment for M abscessus pulmonary disease. These were among findings of a recent study published in the journal Chest.

In the current study, researchers sought to evaluate the safety and outcomes associated with compassionate use of ALIS in M abscessus pulmonary disease in 41 patients from 5 different countries. Through the Nontuberculous Mycobacteria Network European Trials Group (NTM-net), physicians who were experienced in the use of ALIS in patients with M abscessus pulmonary disease were recruited for the current analysis. Pseudonymization information from the 41 participants was obtained via an online case report form. All treatment outcomes were defined based on the statement from the NTM-net, with the outcomes of cure, microbiological cure, and clinical cure combined into “good outcome.” In addition, drug susceptibility was defined according to international guidelines.

Among the 41 enrolled patients, cystic fibrosis (CF) and non-CF bronchiectasis were the most common predisposing disorders, reported in 51.2% and 31.7% of participants, respectively. The most prevalent disease manifestation was nodular-bronchiectatic  disease, which was reported in 73.1% of patients. Overall, 61% of patients were infected with M abscessus subspecies abscessus, with 6 of the isolates (14.6%) amikacin-resistant and 56.1% macrolide-resistant. Treatment with ALIS was initiated because of toxicity associated with intravenous (IV) amikacin in 24.4% (10 of 41) of patients, to strengthen the oral regimen of amikacin in the continuation phase in 14.6% (6 of 41) of patients, for the treatment of refractory M abscessus disease in 19.5% (8 of 41) of patients, and for other/unknown reasons in 41.5% (17 of 41) of patients.

IV amikacin–related toxicity included ototoxicity in 8 participants, renal toxicity in 1 participant, and unspecified toxicity in 1 participant. In 1 of the patients with IV amikacin–associated ototoxicity, treatment with ALIS was also linked to the development of ototoxicity. Overall, culture conversion was achieved in 43.9% of the participants. A good outcome was reported in 61.0% (25 of 41) of the patients. Treatment failure was observed in 13 participants, death was reported in 2 participants, and the outcome was unknown in 1 of the participants.

Of the 6 patients who reported amikacin-resistant isolates, 2 (33.3%) experienced a good outcome. There were no differences with respect to good outcome observed between macrolide-susceptible and macrolide-resistant strains. In patients with CF, a trend toward poorer treatment outcomes was observed compared with non-CF patients (47.6% vs 75.0%, respectively; P =.069).

Adverse events associated with the administration of ALIS were reported in 65.9% of participants, with the most commonly reported AEs including cough in 43.9% of patients, dyspnea in 22.0%, and ototoxicity in 22.0%. Adverse events were linked to treatment discontinuation in 14.6% (6 of 41) of patients.

The investigators concluded that the data derived from this study must be interpreted with caution, because of the small, albeit heterogeneous, cohort. Also, ALIS was incorporated rather late in the treatment regimen because of limited access. Thus, the efficacy of ALIS for the treatment of M abscessus pulmonary disease cannot be easily estimated from the current analysis. According to these results, a clinical trial with an ALIS-containing regimen for M abscessus pulmonary disease is warranted, with its primary role possibly being in the continuation phase of treatment — that is, as a companion to 2 or fewer active oral antibiotics.

Disclosure: Some of the study authors have declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures. 


Sanne MHZ, Chiron R, Essink S, et al. Safety and outcomes of amikacin liposome inhalation suspension for Mycobacterium abscessus pulmonary disease: a NTM-NET study. Chest. Published online January 18, 2022.  doi:10.1016/j.chest.2022.01.015