Outcome Measures for Clinical Trials in Interstitial Lung Diseases

Title:Outcome Measures for Clinical Trials in Interstitial Lung Diseases

Volume: 11 Issue: 2

Author(s): Matthew R. Lammi, Robert P. Baughman, Surinder S. Birring, Anne-Marie Russell, Jay H. Ryu, Mary Beth Scholand, Oliver Distler, Daphne LeSage, Catherine Sarver, Katerina Antoniou, Kristin B Highland, Otylia Kowal-Bielecka, Joseph A. Lasky, Athol U. Wells and Lesley Ann Saketkoo

Affiliation:

Keywords: Chronic fibrosing, connective tissue disease related interstitial lung disease, idiopathic interstitial pneumonia, idiopathic pulmonary fibrosis, rheumatoid arthritis, scleroderma, systemic sclerosis.

Abstract: The chronic fibrosing idiopathic interstitial pneumonias (IIPs) are a group of heterogeneous pulmonary parenchymal disorders described by radiologic and histological patterns termed usual interstitial pneumonia (UIP) and non-specific interstitial pneumonia (NSIP). These include idiopathic pulmonary fibrosis (IPF) and those related to connective tissue disease (CTD) and are associated with substantial morbidity and mortality. Beyond the importance of establishing an appropriate diagnosis, designing optimal clinical trials for IIPs has been fraught with difficulties in consistency of clinical endpoints making power analyses, and the establishment of efficacy and interpretation of results across trials challenging.

Preliminary recommendations, developed by rigorous consensus methods, proposed a minimum set of outcome measures, a ‘core set’, to be incorporated into future clinical trials (Saketkoo et al, THORAX. 2014.). This paper sets out to examine the candidate instruments for each domain (Dyspnea, Cough, Health Related Quality of Life, Imaging, Lung Physiology and Function, Mortality). Candidate measures that were not selected as well as measures that were not available for examination at the time of the consensus process will also be discussed.

Cite this article as:

Lammi R. Matthew, Baughman P. Robert, Birring S. Surinder, Russell Anne-Marie, Ryu H. Jay, Scholand Beth Mary, Distler Oliver, LeSage Daphne, Sarver Catherine, Antoniou Katerina, Highland B Kristin, Kowal-Bielecka Otylia, Lasky A. Joseph, Wells U. Athol and Saketkoo Ann Lesley, Outcome Measures for Clinical Trials in Interstitial Lung Diseases, Current Respiratory Medicine Reviews 2015; 11 (2) . https://dx.doi.org/10.2174/1573398X11666150619183527