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Outcomes and endpoints reported in studies of pulmonary exacerbations in people with cystic fibrosis: A systematic review

There is no consensus about which outcomes should be evaluated in studies of pulmonary exacerbations in people with cystic fibrosis (CF). Outcomes used for evaluation should be meaningful; that is, they should capture how people feel, function or survive and be acknowledged as important to people with CF, or should be reliable surrogates of those outcomes. We aimed to summarise the outcomes and corresponding endpoints which have been reported in studies of pulmonary exacerbations, and to identify those which are most likely to be meaningful.

Citation:
McLeod C, Wood J, Schultz A, Norman R, Smith S, Blyth CC, et al. Outcomes and endpoints reported in studies of pulmonary exacerbations in people with cystic fibrosis: A systematic review. J Cyst Fibros. 2020;19(6):858-67.

Keywords:
Cystic fibrosis; Endpoint measure; Outcome assessment; Outcome variables; Patient reported outcome measures; Pulmonary exacerbation.

Abstract:
There is no consensus about which outcomes should be evaluated in studies of pulmonary exacerbations in people with cystic fibrosis (CF). Outcomes used for evaluation should be meaningful; that is, they should capture how people feel, function or survive and be acknowledged as important to people with CF, or should be reliable surrogates of those outcomes. We aimed to summarise the outcomes and corresponding endpoints which have been reported in studies of pulmonary exacerbations, and to identify those which are most likely to be meaningful.