Scope and Consistency of Outcomes Reported in Trials of Patients With Systemic Sclerosis.

Abstract

The core outcome set for trials in systemic sclerosis (SSc) was developed in 2008 and comprises 11 domains and 31 measures, leading to the development of the Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS). We aimed to assess the scope and consistency of outcomes reported in trials of SSc and the uptake of this core set and the CRISS. Medline, the Cochrane Central Register of Controlled Trials, Embase, and ClinicalTrials.gov were searched to identify randomized trials published from January 1, 2000 to April 29, 2018 in adults with limited or diffuse SSc. Outcomes and measures were recorded for each trial, classified into domains and the frequency of outcomes before after publication of the publication of the core set calculated. From 152 trials, 4,193 outcomes were classified into 84 domains. The 3 most common domains were health-related quality of life (HRQoL) and function (59%, 130 measures), skin (47%, 59 measures), and pulmonary (45%, 168 measures). After the publication of the core outcome set, no trial reported the complete core set with adherence to each of the 11 domains, ranging from 6.1% to 54.4% and adherence to each of the 31 measures ranging from 0% to 48.1%. The 5 measures required for the CRISS were reported completely in 11% of trials. Despite recognition that uniform acquisition and reporting of outcomes would enable a better evaluation of proposed SSc therapeutics, the outcome domains and measures reported in randomized trials in SSc remain very inconsistent, with little impact of the core outcome set.