Composite outcomes in observational studies of ulcerative colitis: A systematic review and meta‐analysis

Abstract

Background: Ulcerative colitis (UC) has been the focus of numerous observational studies over the years and a common strategy employed in their design is the use of composite and aggregate outcomes.Objective: This systematic review and meta-analysis aims to identify composite and aggregate outcomes of observational studies in UC and to evaluate how the number and type of variables included and the length of follow-up affect the frequency of patients that achieve these outcomes.Methods: A systematic literature search was carried out using MEDLINE [via PubMed], Scopus, and Web of Science online databases. Observational studies that included UC patients and reported composite or aggregate outcomes were identified. A set of variables considered to be representative of progressive or disabling UC was defined, the proportion of patients attaining the outcomes was determined and a random-effects meta-analysis was performed by dividing the identified studies into subgroups according to different criteria of interest.Results: A total of 10,264 records were identified in the systematic search, of which 33 were retained for qualitative analysis and 20 were included in the meta-analysis.The mean frequency for composite outcomes was 0.363 [95% confidence interval (CI) 0.323-0.403]. The frequency of composite outcome for the subgroup of studies that included the variable "Biologics" was significantly higher than for those in which this variable was not reported [0.410; 95% CI 0.364-0.457 versus 0.298; 95% CI 0.232-0.364; p = 0.006]. Composite outcomes were also more frequent as the follow-up duration increased. Conclusion:The frequency of composite outcomes in observational studies of UC is dependent on the specific identity of the variables being reported. Moreover, longer

How many biomarker measurements are needed to predict prognosis in Crohn's disease patients under infliximab?—A prospective study

“…The second outcome (composite outcome 2) was identical to the first except for the exclusion of IFX dose escalation or interval reduction as one of the individual items. These composite endpoints were adapted from previous studies 9,12 …”

“…Two composite outcomes were used, both reflecting disease pro- were adapted from previous studies. 9,12…”

“…In the present study, we intended to evaluate the performance of easily available biomarker tests in the prediction of CD prognosis, evaluated through composite outcomes. A combination of individual variables allows for the optimization of resources and increases the accuracy and statistical power, and therefore composite outcomes have been increasingly favored in both interventional and observational studies aimed at exploring the effectiveness of a given therapeutic strategy 9 . We performed an analysis of the data from the DIRECT, a 2‐year, prospective multicenter study, with the primary aim of identifying the most relevant parameters for the prediction of CD progression in patients under maintenance therapy with infliximab.…”

Mild to moderate ulcerative colitis: Call me by my name

“…Nevertheless, the most recent clinical trials assessing drug efficacy in UC have already incorporated new items among their outcomes, both isolated or in combination 7 . For instance, certain biomarkers (e.g., fecal calprotectin) are now considered formal targets of medical treatment, along with endoscopic healing, quality of life, and disability.…”

“…Nevertheless, the most recent clinical trials assessing drug efficacy in UC have already incorporated new items among their outcomes, both isolated or in combination. 7 For instance, certain biomarkers (e.g., fecal calprotectin) are now considered formal targets of medical treatment, along with endoscopic healing, quality of life, and disability. Meanwhile, the evaluation of histological disease activity has been reported with the newly developed compounds, but it is still not considered a formal target in the STRIDE‐II recommendations.…”