Gastrointestinal Impact and Tool Performance in Juvenile Systemic Sclerosis Using the University of California, Los Angeles Scleroderma Clinical Trial Consortium Gastrointestinal Tract, Version 2.0 Assessment.

Abstract

The objective of this study is to characterize gastrointestinal (GI) manifestations in patients with juvenile-onset systemic sclerosis (jSSc) using the University of California, Los Angeles Scleroderma Clinical Trial Consortium Gastrointestinal Tract, version 2.0 (UCLA GIT 2.0) patient-reported outcome (PRO) instrument, and to evaluate its validity and responsiveness in this population. Patients with jSSc from the National Registry for Childhood Onset Scleroderma who completed the UCLA GIT 2.0 were included. Demographic and clinical data, domain, and total UCLA GIT 2.0 scores were summarized. Convergent validity was assessed by Spearman correlations with the Scleroderma Health Assessment Questionnaire GI visual analog scale (SHAQ-GI-VAS) and the SHAQ Disease VAS (SHAQ-DIS-VAS). Responsiveness was explored in patients with paired UCLA GIT 2.0 assessments one year later. Fifty-one patients with jSSc (mean age at onset, 9.8 years; mean disease duration, 4.4 years) had a mean UCLA GIT 2.0 total score of 0.30, indicating a mild GI burden. Distension and bloating and reflux were the most affected domains, each reported in more than 70% of patients. Total and subscale UCLA GIT 2.0 scores showed moderate to strong significant correlations with the SHAQ-GI-VAS and SHAQ-DIS-VAS, supporting convergent validity. Among 22 patients with paired data, the mean total UCLA GIT 2.0 improved by 0.11 points (P = 0.039), and 55% of these patients achieved a clinically important improvement in at least one domain, indicating preliminary responsiveness. The UCLA GIT 2.0 captures the frequency and severity of GI symptoms in patients with jSSc and demonstrates acceptable validity and sensitivity to change. Although developed for adults, the instrument appears suitable for monitoring GI outcomes in pediatric patients with SSc in both research and clinical settings.