title: Outcome Measures for Clinical Trials in Interstitial Lung Diseases.
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.
authors.affiliation: Louisiana State University Health Sciences Center, New Orleans, New Orleans, LA, USA; New Orleans Scleroderma and Sarcoidosis Patient Care and Research Center; New Orleans, LA, USA.
authors.forename: Matthew R
authors.initials: MR
authors.lastname: Lammi
authors.affiliation: University of Cincinnati, OH, USA.
authors.forename: Robert P
authors.initials: RP
authors.lastname: Baughman
authors.affiliation: Division of Asthma, Allergy and Lung Biology, King's College London, UK.
authors.forename: Surinder S
authors.initials: SS
authors.lastname: Birring
authors.affiliation: Royal Brompton Hospital and National Heart and Lung Institute; London, UK.
authors.forename: Anne-Marie
authors.initials: AM
authors.lastname: Russell
authors.affiliation: Mayo Clinic College of Medicine, Rochester, MN, USA.
authors.forename: Jay H
authors.initials: JH
authors.lastname: Ryu
authors.affiliation: University of Utah, Salt Lake City, UT, USA.
authors.forename: Marybeth
authors.initials: M
authors.lastname: Scholand
authors.affiliation: Division of Rheumatology, University Hospital Zurich, Switzerland.
authors.forename: Oliver
authors.initials: O
authors.lastname: Distler
authors.affiliation: Patient Research Partner, Office of Public Health, New Orleans, LA, USA.
authors.forename: Daphne
authors.initials: D
authors.lastname: LeSage
authors.affiliation: Patient Research Partner, Maryland, USA.
authors.forename: Catherine
authors.initials: C
authors.lastname: Sarver
authors.affiliation: University of Crete, Heraklion, Greece.
authors.forename: Katerina
authors.initials: K
authors.lastname: Antoniou
authors.affiliation: Cleveland Clinic Foundation, Respiratory Institute, Cleveland, OH, USA.
authors.forename: Kristin B
authors.initials: KB
authors.lastname: Highland
authors.affiliation: Medical University of Bialystok, Bialystok, Poland.
authors.forename: Otylia
authors.initials: O
authors.lastname: Kowal-Bielecka
authors.affiliation: New Orleans Scleroderma and Sarcoidosis Patient Care and Research Center; New Orleans, LA, USA; Tulane University Lung Center; New Orleans, LA, USA.
authors.forename: Joseph A
authors.initials: JA
authors.lastname: Lasky
authors.affiliation: Royal Brompton Hospital and National Heart and Lung Institute; London, UK.
authors.forename: Athol U
authors.initials: AU
authors.lastname: Wells
authors.affiliation: New Orleans Scleroderma and Sarcoidosis Patient Care and Research Center; New Orleans, LA, USA; Tulane University Lung Center; New Orleans, LA, USA.
authors.forename: Lesley Ann
authors.initials: LA
authors.lastname: Saketkoo
keywords: Chronic fibrosing; connective tissue disease related interstitial lung disease; idiopathic interstitial pneumonia; idiopathic pulmonary fibrosis; rheumatoid arthritis; scleroderma; systemic sclerosis