Table 1A.

Summary of case studies.

Case StudyFull TitleWhat are the outcome domains you are currently working with?Why have you chosen them?What contextual factors did you consider?How did you make these decisions?
CTD-ILDConnective tissue diseases — interstitial lung diseaseDyspnea; health related quality of life; lung imaging; lung physiology and function; survival; coughThese domains have been identified as the most relevant and measureable for clinical trials in CTD-ILD and interstitial pulmonary fibrosis by medical and patient expertsPrimary context is the randomized clinical trial; also considered was the context of clinical relevance of the domains to patient outcome in usual clinical careInformed by expert Delphi involving rheumatologists and pulmonologists; a 3-round Delphi identified potential domains and measures; patient perspective solicited via survey and patient focus groups
PMRPolymyalgia rheumaticaPain; stiffness; function; systemic inflammationCandidate outcome measures chosen for a postulated future interventional trial of an alternative to prednisolone for PMRAge; gender; cultural background; time of day specified in the patient-reported outcome measuresInformal patient consultation; systematic literature review; work in progress
VasculitisANCA-associated vasculitisDisease activity; disease damage; patient-reported outcome; mortalityThese domains have been measured in many trials and have been considered critical for both evaluating efficacy and guiding evaluation and treatmentPrimary context was the randomized clinical trial; trials have often been modeled on standard of clinical careExtrapolation from clinical trials; expert opinion; patient surveys