Pneumologie 2016; 70 - A8
DOI: 10.1055/s-0036-1592232

12-Month Follow-up From Phase 2 Trial of Liposomal Amikacin for Inhalation (LAI) in Patients With Nontuberculous Mycobacterial (NTM) Lung Infection

K Olivier 1, D Griffith 2, K Winthrop 3, B Brown-Elliott 2, G Eagle 4, J McGinnis 4, R Wallace 2
  • 1National Institutes of Health
  • 2University of Texas Health Science Center
  • 3Oregon Health & Science University
  • 4Insmed Incorporated

Introduction:

TR02 – 112 enrolled adult patients with NTM lung infection due to MAC and/or Mycobacterium abscessus refractory to a multi-drug regimen for ≥6 months. The randomized, placebo-controlled study evaluated efficacy, safety, and tolerability of LAI 590 mg administered once daily added to multi-drug regimen for 84 days (Double-blind). Patients continuing in the Open-label phase received LAI added to multi-drug regimen for 84 additional days. Here we report long term safety and efficacy data collected for patients in the 12 months after the last dose of LAI.

Methods:

The follow-up period was included to allow long term LAI safety and efficacy assessments 12 months after the last dose of LAI.

Data collected included a review of medical conditions and hospitalizations, current medications, vital signs, physician's assessment, and sputum samples for microbiology.

Results:

Sixty-five patients entered the long-term follow-up. Fifty-seven patients completed the 12-month follow up visit. Seven patients died during the follow-up period. Only 1 of these patients achieved culture conversion (3 consecutive negative sputum cultures) during the treatment phase. 'Pulmonary exacerbations' were the most frequent medical condition at the follow-up visit. No clinically meaningful changes from baseline in vital signs or physical examination were observed. Among patients with available follow-up data, those who culture-converted during either treatment phase were more likely to demonstrate negative sputum cultures at the 12-month follow-up vs. those who did not (14/17; 82.5% vs. 6/28; 21.4%, respectively).

Conclusion:

For many patients achieving culture conversion after 3 – 6 months of add-on LAI treatment resulted in sustained negative sputum cultures 12 months after LAI was discontinued. Study findings confirm the durability of LAI treatment response and continue to support the potential role of LAI as a viable add-on treatment to standard multi-drug regimens for NTM lung infections in patients' refractory to previous regimens.