Abstract
Amikacin liposome inhalation suspension (ALIS) [Arikayce(®) Liposomal (EU); Arikayce(®) (USA)], a liposomal suspension of the aminoglycoside amikacin (590 mg) for nebulization via the Lamira(®) Nebulizer System, is available as add-on therapy for treatment-refractory Mycobacterium avium complex (MAC) lung disease in adults who have little or no alternative treatment options. Its addition to guideline-based therapy (GBT) significantly improved the likelihood of achieving sputum culture conversion (defined as three consecutive monthly MAC-negative sputum cultures) by month 6 relative to GBT alone in adults with treatment-refractory MAC lung disease, with the conversion response maintained over up to 12 months' therapy and at 3 months' post treatment in significantly higher proportions of ALIS plus GBT than GBT alone recipients. ALIS as an add-on therapy to GBT was associated with an increased risk of respiratory adverse reactions compared with GBT alone, but treatment-emergent adverse events associated with systemic amikacin exposure were uncommon.