RT Journal Article
SR Electronic
T1 Connective tissue disease related interstitial lung diseases and idiopathic pulmonary fibrosis: provisional core sets of domains and instruments for use in clinical trials
JF Thorax
JO Thorax
FD BMJ Publishing Group Ltd and British Thoracic Society
SP 436
OP 444
DO 10.1136/thoraxjnl-2013-204202
VO 69
IS 5
A1 Lesley Ann Saketkoo
A1 Shikha Mittoo
A1 Dörte Huscher
A1 Dinesh Khanna
A1 Paul F Dellaripa
A1 Oliver Distler
A1 Kevin R Flaherty
A1 Sid Frankel
A1 Chester V Oddis
A1 Christopher P Denton
A1 Aryeh Fischer
A1 Otylia M Kowal-Bielecka
A1 Daphne LeSage
A1 Peter A Merkel
A1 Kristine Phillips
A1 David Pittrow
A1 Jeffrey Swigris
A1 Katerina Antoniou
A1 Robert P Baughman
A1 Flavia V Castelino
A1 Romy B Christmann
A1 Lisa Christopher-Stine
A1 Harold R Collard
A1 Vincent Cottin
A1 Sonye Danoff
A1 Kristin B Highland
A1 Laura Hummers
A1 Ami A Shah
A1 Dong Soon Kim
A1 David A Lynch
A1 Frederick W Miller
A1 Susanna M Proudman
A1 Luca Richeldi
A1 Jay H Ryu
A1 Nora Sandorfi
A1 Catherine Sarver
A1 Athol U Wells
A1 Vibeke Strand
A1 Eric L Matteson
A1 Kevin K Brown
A1 James R Seibold
YR 2014
UL http://thorax.bmj.com/content/69/5/436.abstract
AB Rationale Clinical trial design in interstitial lung diseases (ILDs) has been hampered by lack of consensus on appropriate outcome measures for reliably assessing treatment response. In the setting of connective tissue diseases (CTDs), some measures of ILD disease activity and severity may be confounded by non-pulmonary comorbidities. Methods The Connective Tissue Disease associated Interstitial Lung Disease (CTD-ILD) working group of Outcome Measures in Rheumatology—a non-profit international organisation dedicated to consensus methodology in identification of outcome measures—conducted a series of investigations which included a Delphi process including >248 ILD medical experts as well as patient focus groups culminating in a nominal group panel of ILD experts and patients. The goal was to define and develop a consensus on the status of outcome measure candidates for use in randomised controlled trials in CTD-ILD and idiopathic pulmonary fibrosis (IPF). Results A core set comprising specific measures in the domains of lung physiology, lung imaging, survival, dyspnoea, cough and health-related quality of life is proposed as appropriate for consideration for use in a hypothetical 1-year multicentre clinical trial for either CTD-ILD or IPF. As many widely used instruments were found to lack full validation, an agenda for future research is proposed. Conclusion Identification of consensus preliminary domains and instruments to measure them was attained and is a major advance anticipated to facilitate multicentre RCTs in the field.