TY - JOUR
T1 - Connective tissue disease related interstitial lung diseases and idiopathic pulmonary fibrosis
T2 - Provisional core sets of domains and instruments for use in clinical trials
AU - Saketkoo, Lesley Ann
AU - Mittoo, Shikha
AU - Huscher, Dörte
AU - Khanna, Dinesh
AU - Dellaripa, Paul F.
AU - Distler, Oliver
AU - Flaherty, Kevin R.
AU - Frankel, Sid
AU - Oddis, Chester V.
AU - Denton, Christopher P.
AU - Fischer, Aryeh
AU - Kowal-Bielecka, Otylia M.
AU - Lesage, Daphne
AU - Merkel, Peter A.
AU - Phillips, Kristine
AU - Pittrow, David
AU - Swigris, Jeffrey
AU - Antoniou, Katerina
AU - Baughman, Robert P.
AU - Castelino, Flavia V.
AU - Christmann, Romy B.
AU - Christopher-Stine, Lisa
AU - Collard, Harold R.
AU - Cottin, Vincent
AU - Danoff, Sonye
AU - Highland, Kristin B.
AU - Hummers, Laura
AU - Shah, Ami A.
AU - Kim, Dong Soon
AU - Lynch, David A.
AU - Miller, Frederick W.
AU - Proudman, Susanna M.
AU - Richeldi, Luca
AU - Ryu, Jay H.
AU - Sandorfi, Nora
AU - Sarver, Catherine
AU - Wells, Athol U.
AU - Strand, Vibeke
AU - Matteson, Eric L.
AU - Brown, Kevin K.
AU - Seibold, James R.
PY - 2014/5
Y1 - 2014/5
N2 - 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.
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.
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U2 - 10.1136/thoraxjnl-2013-204202
DO - 10.1136/thoraxjnl-2013-204202
M3 - Article
C2 - 24368713
AN - SCOPUS:84898849450
SN - 0040-6376
VL - 69
SP - 428
EP - 436
JO - Thorax
JF - Thorax
IS - 5
ER -