Current Respiratory Medicine Reviews

Joseph Varon  
The University of Texas Health Science Center
Houston, TX
USA

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Outcome Measures for Clinical Trials in Interstitial Lung Diseases

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, Lesley Ann Saketkoo.

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.

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

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Article Details

VOLUME: 11
ISSUE: 2
Year: 2015
Page: [163 - 174]
Pages: 12
DOI: 10.2174/1573398X11666150619183527
Price: $58