P4.22: Reporting on outcome measures in pediatric chronic intestinal failure: a systematic review

Abstract

Introduction: Standardized outcome measures provide the ability of comparing outcomes of different clinical trials. Furthermore, standardized outcome measures offer the possibility of reducing potential reporting bias and increase the reporting of patient relevant outcomes. The aim of this study is to systematically assess how definitions and outcome measures are defined in therapeutic trials of children with chronic Intestinal Failure (IF). These results will be used as first step in the development of a Core Outcome Set (COS). Methods: MEDLINE, EMBASE, and Cochrane Central Register of Controlled Trials (CENTRAL) databases were searched from inception till august 2018. Studies were included if it was an original interventional study or observational study with a control group, concerning children from 0–18 years of age old with chronic IF and concomitant parenteral nutrition use, and a definition of IF was provided. No language restriction was used. Outcomes were mapped to one of the five core areas from the OMERACT 2.0 filter. The Delphi List, Newcastle Ottawa Scale for non-randomized studies, or the quality assessment tool for before-after studies (National Heart, Lung, and Blood Institute) were used to assess the quality of included studies. Results: A total of 1766 articles were found of which 63 studies fulfilled our inclusion criteria (figure 1). 47 studies (75%) did not report any definition of IF. Of the 16 studies (25%) which reported a definition of IF, 5 different definitions were found. A total of 106 different outcomes were reported. The four most reported outcome measures were: mortality (n=23), growth (n=22), liver enzymes (n=18), and parenteral nutrition weaning (n=18). The majority (n=63; 59%) of reported outcomes was mapped to the pathophysiological manifestations core area from the OMERACT 2.0 filter. 17 studies (27%) predefined a primary outcome of which central line related infection was the most frequently used (n=5). Quality of reporting was considered low in most studies (n=47; 75%). Conclusion: Heterogeneity exists in the definitions of IF, and outcome reporting in research concerning pediatric chronic IF.