title: Connective tissue disease related interstitial lung diseases and idiopathic pulmonary fibrosis: provisional core sets of domains and instruments for use in clinical trials.
abstract: BACKGROUND
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Louisiana State University Health Sciences Center, , New Orleans, Louisiana, USA.
authors.forename: Lesley Ann
authors.initials: LA
authors.lastname: Saketkoo
authors.forename: Shikha
authors.initials: S
authors.lastname: Mittoo
authors.forename: Dörte
authors.initials: D
authors.lastname: Huscher
authors.forename: Dinesh
authors.initials: D
authors.lastname: Khanna
authors.forename: Paul F
authors.initials: PF
authors.lastname: Dellaripa
authors.forename: Oliver
authors.initials: O
authors.lastname: Distler
authors.forename: Kevin R
authors.initials: KR
authors.lastname: Flaherty
authors.forename: Sid
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authors.lastname: Frankel
authors.forename: Chester V
authors.initials: CV
authors.lastname: Oddis
authors.forename: Christopher P
authors.initials: CP
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authors.forename: Aryeh
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authors.lastname: Fischer
authors.forename: Otylia M
authors.initials: OM
authors.lastname: Kowal-Bielecka
authors.forename: Daphne
authors.initials: D
authors.lastname: LeSage
authors.forename: Peter A
authors.initials: PA
authors.lastname: Merkel
authors.forename: Kristine
authors.initials: K
authors.lastname: Phillips
authors.forename: David
authors.initials: D
authors.lastname: Pittrow
authors.forename: Jeffrey
authors.initials: J
authors.lastname: Swigris
authors.forename: Katerina
authors.initials: K
authors.lastname: Antoniou
authors.forename: Robert P
authors.initials: RP
authors.lastname: Baughman
authors.forename: Flavia V
authors.initials: FV
authors.lastname: Castelino
authors.forename: Romy B
authors.initials: RB
authors.lastname: Christmann
authors.forename: Lisa
authors.initials: L
authors.lastname: Christopher-Stine
authors.forename: Harold R
authors.initials: HR
authors.lastname: Collard
authors.forename: Vincent
authors.initials: V
authors.lastname: Cottin
authors.forename: Sonye
authors.initials: S
authors.lastname: Danoff
authors.forename: Kristin B
authors.initials: KB
authors.lastname: Highland
authors.forename: Laura
authors.initials: L
authors.lastname: Hummers
authors.forename: Ami A
authors.initials: AA
authors.lastname: Shah
authors.forename: Dong Soon
authors.initials: DS
authors.lastname: Kim
authors.forename: David A
authors.initials: DA
authors.lastname: Lynch
authors.forename: Frederick W
authors.initials: FW
authors.lastname: Miller
authors.forename: Susanna M
authors.initials: SM
authors.lastname: Proudman
authors.forename: Luca
authors.initials: L
authors.lastname: Richeldi
authors.forename: Jay H
authors.initials: JH
authors.lastname: Ryu
authors.forename: Nora
authors.initials: N
authors.lastname: Sandorfi
authors.forename: Catherine
authors.initials: C
authors.lastname: Sarver
authors.forename: Athol U
authors.initials: AU
authors.lastname: Wells
authors.forename: Vibeke
authors.initials: V
authors.lastname: Strand
authors.forename: Eric L
authors.initials: EL
authors.lastname: Matteson
authors.forename: Kevin K
authors.initials: KK
authors.lastname: Brown
authors.forename: James R
authors.initials: JR
authors.lastname: Seibold
mesh_terms: D003226:Congresses as Topic; D003240:Connective Tissue Diseases / Q000175:diagnosis / Q000628:therapy*; D032921:Consensus; D006801:Humans; D054990:Idiopathic Pulmonary Fibrosis / Q000175:diagnosis / Q000628:therapy*; D007391:International Cooperation; D017563:Lung Diseases, Interstitial / Q000175:diagnosis / Q000628:therapy*; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D012042:Registries; D012955:Societies, Medical
keywords: Connective tissue disease associated lung disease; Idiopathic pulmonary fibrosis; Interstitial Fibrosis; Rheumatoid lung disease; Systemic disease and lungs