Exploring the outcomes and endpoints used in gastrointestinal research in cystic fibrosis: a systematic review

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Abstract Background Cystic fibrosis (CF) research has increasingly focused on understanding the extra-pulmonary manifestations of CF, including on the gastrointestinal (GI) system. The effect of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies outside the lungs is also a topic of research interest and both are key research priorities. However, significant evidence gaps persist in understanding the complex pathophysiology of CFTR dysfunction in the GI tract, and the treatment of these GI problems. Inconsistencies in outcome reporting may contribute towards these evidence gaps, and a standardised approach to outcome reporting may help to address this. This systematic rapid review aims to identify and catalogue the range of outcome measurement instruments (OMIs) and associated endpoints currently used in CF GI research. Methods This PROSPERO-registered review (CRD42021281961) was conducted following Cochrane Rapid Reviews Methods Group and COMET initiative guidance. Comprehensive searches were performed in MEDLINE, EMBASE, PubMed, Cochrane Library, and ongoing clinical trials databases, covering an 11-year period (August 2013 to November 2024). Screening and data extraction were carried out using Covidence online software. Results A total of 1,541 studies were identified, of which 193 met inclusion criteria. These studies collectively used 246 distinct OMIs, of which 172 (70%) were employed in only one study. The OMIs identified were grouped into 14 sub-domains representing key areas of GI research in CF, which were subsequently mapped to 11 of the 38 outcome domains in the taxonomy proposed by the COMET Initiative. The identified outcomes spanned a diverse range of mechanistic and patient-centred measures, reflecting the complexity of GI disease in CF. Conclusions Current research into the GI tract in CF uses a heterogeneous array of OMIs, with limited standardisation. This highlights both the complexity of CFTR dysfunction within the GI tract, requiring a wide scope of OMIs to address this, as well the variability and potential inefficiency in current outcome reporting practices. To advance our understanding of CF pathophysiology in the GI tract, a standardised approach to outcome reporting is needed. Our findings support the development of a core outcome set to promote reporting consistency and improve comparability across studies in CF GI research.

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