Sustainability and Durability of Culture Conversion in Patients Receiving Amikacin Liposome Inhalation Suspension (ALIS) for Treatment-Refractory Mycobacterium Avium Complex Lung Disease (MAC-LD) in the CONVERT Study

Introduction: Treatment options for refractory MAC-LD are limited. By month 6 in CONVERT, culture conversion rates in adults with refractory MAC-LD were 29.0% (65/224) with ALIS, a nebulized, liposome-encapsulated amikacin formulation, + guideline-based therapy (GBT) vs 8.9% (10/112; P Aims: We report safety, culture conversion durability, and functional response (6-minute walk test; 6MWT) with extended (up to 16 mo) ALIS therapy. Methods: In CONVERT, patients were randomly assigned to receive ALIS 590mg QD + GBT or GBT alone. Patients with culture conversion (3 consecutive monthly MAC-negative sputum cultures) by month 6 continued treatment for 12 months after the time of culture conversion. Results: In patients who converted by month 6 and continued treatment, 63% (41/65) of those on ALIS+GBT and 30% (3/10) on GBT alone remained culture negative after 12 months of subsequent treatment; 63% (41/65) vs 0% (0/10) remained culture-negative 3 months off all antibiotics. There was no difference between treatment arms in the change in 6MWT distance from baseline to month 8 (P=0.84). Extended ALIS exposure did not alter overall safety findings (Griffith, AJRCCM 2018) with ALIS+GBT and GBT alone; respectively, 84.7% (195/223) and 50.9% (57/112) had respiratory treatment-emergent adverse events (TEAEs); 20.2% (45/223) and 20.5% (23/112) had serious TEAEs. Conclusion: Addition of ALIS to GBT in patients with refractory MAC-LD improved culture conversion, which was maintained during therapy and for 3 months posttreatment. No new safety signals emerged with extended treatment.