Gastrointestinal Impact and Tool Performance in Juvenile Systemic Sclerosis Using the University of California, Los...

Area of Science:

• Pediatric Rheumatology
• Gastroenterology
• Clinical Outcomes Assessment

Background:

• Juvenile-onset systemic sclerosis (jSSc) frequently involves gastrointestinal (GI) complications.
• Accurate assessment tools are crucial for monitoring GI manifestations in pediatric SSc patients.

Purpose of the Study:

• To characterize GI symptoms in jSSc using the UCLA Scleroderma Clinical Trial Consortium Gastrointestinal Tract 2.0 (UCLA GIT 2.0) instrument.
• To evaluate the validity and responsiveness of the UCLA GIT 2.0 in a pediatric SSc population.

Main Methods:

• Utilized data from 51 jSSc patients from the National Registry for Childhood Onset Scleroderma.
• Assessed convergent validity against SHAQ-GI-VAS and SHAQ-DIS-VAS.
• Explored responsiveness using paired assessments over one year.

Main Results:

• The mean UCLA GIT 2.0 Total score was 0.30, indicating mild GI burden.
• Distension/bloating and reflux were the most prevalent GI symptoms (>70% of patients).
• The instrument demonstrated significant correlations with other measures (supporting validity) and showed improvement over time (indicating responsiveness).

Conclusions:

• The UCLA GIT 2.0 is a valid and sensitive tool for assessing GI symptoms in jSSc.
• The instrument is suitable for monitoring GI outcomes in pediatric SSc in research and clinical practice.