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.
To determine whether a core set of outcome measures that are reliable and feasible can be identified by experts for use in future clinical trials in connective tissue disease associated interstitial lung disease (CTD-ILD) and idiopathic pulmonary fibrosis (IPF).
ContributorsSaketkoo, Lesley Ann Mittoo, Shikha Huscher, Dorte Khanna, Dinesh Dellaripa, Paul F. Distler, Oliver Flaherty, Kevin R. Frankel, Sid Oddis, Chester V. Denton, Christopher P. Fischer, Aryeh Kowal-Bielecka, Otylia M. LeSage, Daphne Merkel, Peter A. Phillips, Kristine Pittrow, David Swigris, Jeffrey Antoniou, Katerina Baughman, Robert P. Castelino, Flavia V. Christmann, Romy B. Christopher-Stine, Lisa Collard, Harold R. Cottin, Vincent Danoff, Sonye Highland, Kristin B. Hummers, Laura Shah, Ami A. Kim, Dong Soon Lynch, David A. Miller, Frederick W. Proudman, Susanna M. Richeldi, Luca Ryu, Jay H. Sandorfi, Nora Sarver, Catherine Wells, Athol U. Strand, Vibeke Matteson, Eric L. Brown, Kevin K. Seibold, James R.
Disease Category: Lungs & airways
Disease Name: Interstitial Lung Diseases
Age Range: Unknown
Sex: Either
Nature of Intervention: Drug
- Clinical experts
- Patient/ support group representatives
- COS for clinical trials or clinical research
- Delphi process
- Focus group(s)
- Nominal group technique (NGT)
- Survey
(1) Medical expert web-based Delphi process; (2) Patient perspective investigations: (a) focus groups with CTD-ILD participants, (b) analysed previously available patient perspective data in IPF patients, (c) CTD-ILD rated on a seven-point Likert scale the importance of the domains identified in medical expert Delphi process; and (3) NGT meeting combining medical expert and patient participants, which lead to identification of preliminary core sets of domains with corresponding instruments that are clinically meaningful and feasible in the context of a 1-year multi-centre RCT for each CTD-ILD and IPF.