Gastrointestinal Impact and Tool Performance in Juvenile Systemic Sclerosis Using the UCLA GIT 2.0 Assessment.

Abstract

Objective: The objective of this study is to characterize gastrointestinal (GI) manifestations in juvenile-onset systemic sclerosis (jSSc) using the UCLA Scleroderma Clinical Trial Consortium Gastrointestinal Tract 2.0 (UCLA GIT 2.0) patient-reported outcome (PRO) instrument, and to evaluate its validity and responsiveness in this population.

Methods: jSSc patients 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 and Global visual analog scales (SHAQ-GI-VAS, SHAQ-DIS-VAS). Responsiveness was explored in patients with paired UCLA GIT 2.0 assessments one year later.

Results: Fifty-one jSSc patients (mean age of onset: 9.8 years; mean disease duration: 4.4 years) had a mean UCLA GIT 2.0 Total score of 0.30, indicating mild GI burden. Distension/bloating and Reflux were the most affected domains, each reported in >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% achieved a clinically important improvement in > 1 domain, indicating preliminary responsiveness.

Conclusion: The UCLA GIT 2.0 captures the frequency and severity of GI symptoms in jSSc and demonstrates acceptable validity and sensitivity to change. Although developed for adults, the instrument appears suitable for monitoring GI outcomes in pediatric SSc in both research and clinical settings.