Patient-reported outcomes improve practice in MAC lung disease

Two patient-reported outcome measures displayed adequate reliability, validity, and responsiveness for the evaluation of respiratory and fatigue symptoms in patients with MAC lung disease. These are the first validated patient-reported outcome tools to assess symptoms in this population, potentially improving clinical practice.

“Mycobacterium avium complex (MAC) is the main cause of non-tuberculous mycobacterial lung disease,” stated Dr Charles Daley (University of Colorado, CO, USA). “There are no properly validated patient- reported outcome measures that assess symptom improvement in patients with MAC lung disease.” The phase 3 ARISE study (NCT04677543) aimed to evaluate the quality- of-life-bronchiectasis respiratory domain (QoL-B RD) and patient-reported outcomes measurement information system-fatigue (PROMIS-F) tools with respect to reliability, validity, and responsiveness [1]. These instruments are used to derive symptom scores in patients with MAC lung disease.

The study included 99 patients with noncavitary MAC lung disease who had not received antibiotics for their current infection. Enrolled participants were randomised 1:1 to treatment with amikacin liposome inhalation suspension (ALIS) or a placebo. Participants in both groups received ethambutol and azithromycin. Dr Daley mentioned that cross-sectional reliability and validity analyses demonstrated the psychometrical robustness of the QoL-B RD and PROMIS-F tools in patients with MAC lung disease. “Empirical cumulative distribution function plots of longitudinal QoL-B RD and PROMIS-F change from baseline to 6 months scores, by patient global impression of severity category, demonstrated consistent separation between patients reporting improved and unchanged respiratory and fatigue symptoms,” elaborated Dr Daley.

The culture conversion rate was significantly higher in the ALIS arm (80.6%) than in the placebo arm (63.9%) at 6 months. Moreover, 74.3% of the participants in the ALIS arm achieved their first negative culture- defining conversion by month 1, whereas 46.7% of the participants in the placebo arm had reached this outcome by month 1. In addition, the median time to first negative culture was 1 month in the ALIS arm and 2 months in the placebo arm. No new safety issues emerged from this study. Dr Daley mentioned that a 15-month confirmatory study is ongoing to further assess the efficacy and safety of ALIS, with 12 months on-treatment and 3 months off-treatment.

 

1. Daley C, et al. A Randomized, Double-blind Trial of Amikacin Liposome Inhalation Suspension in Adults With Newly Diagnosed or Recurrent Mycobacterium Avium Complex Lung Disease to Validate Patient-reported Outcome Instruments and Assess Microbiological Outcomes of Treatment: The ARISE Study. Breaking News: 2024 Clinical Trial Results in Pulmonary Medicine. ATS 2024, 17–22 May, San Diego, USA.

Medical writing support was provided by Robert van den Heuvel.

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Posted on 10 June 202410 June 2024