Background/Purpose: Gastrointestinal (GI) tract involvement is nearly universal in patients with systemic sclerosis (SSc) leading to significant morbidity and mortality. In a North American juvenile SSc (jSSc) registry, GI involvement had the strongest correlation with poorer quality of life (QoL) of any organ system. Esophageal dysmotility is the most common GI manifestation and can lead to moderate to severe malnutrition (identified in 14% of jSSc patients, Stevens et al, 2018). Despite the known prevalence and impact of GI tract pathology in SSc, validated outcomes measures are lacking. The International Juvenile Systemic Sclerosis Outcome Group is working to generate consensus organ-specific outcome measures for use in future jSSc studies. We report findings from our GI scoping literature review with a focus on esophageal measures.

Methods: From 4 databases, 24,849 studies were screened, yielding 277 studies identified by title and abstract of which 134 full text articles were extracted (Figure 1). Each level of screening and extraction required two reviewers. A pediatric gastroenterologist experienced in jSSc care helped guide extractions. Twenty-six GI measures were identified and sorted by GI region studied. Extracted data included the presence of jSSc patients, significant change in the outcome measure over time or following intervention, ability of the measure to differentiate SSc from healthy controls (HC), and correlation of the measure with symptoms and QoL measures.

Results: Of 134 studies, only 3.7% were randomized control trials (total of 4 GI measures evaluated). Only 2.2% of the studies included pediatric patients. The most commonly studied GI region was the esophagus (64.9%) followed by stomach (20.9%), colorectum (20.1%), small intestine (15.7%), and oropharynx (7.5%). Nutritional outcomes were reported in 11.9% while QoL measures were included in 31.3% of studies (Table 1). Table 2 shows the characteristics of esophageal measures in SSc. 50/87 (57%) esophageal studies reported on esophageal manometry, with endoscopy the next most commonly reported esophageal measure (25/87, 29%). Esophageal manometry was accurately able to differentiate SSc from HC. Very few studies assessed change over time or in response to intervention. Only 4 of the 26 measures (15%) have been studied in jSSc (esophageal manometry, pH probe, chest CT and esophagram/Upper GI). In studies that evaluated the correlation between GI measures and GI symptoms, correlation was inconsistent.

Conclusion: Esophageal manometry is the most widely studied GI outcome measure in SSc. Despite its established use in children with other GI motility disorders (e.g., achalasia), there were no studies that evaluated esophageal manometry in patients with jSSc. Overall, only 2% of the studies included pediatric patients, and < 10% of studies evaluated the ability of GI outcome measures to assess response to intervention or change over time. Nearly all measures studied demonstrated inconsistent correlation with GI symptoms. Given the morbidity and impact on QoL associated with GI involvement, our study highlights the need for more studies that evaluate GI involvement in SSc, especially for jSSc.

Figure 1: PRISMA systematic review flow diagram

Table 1: Characteristics of SSc Studies on GI Outcome Measures from Scoping Review

Table 2: Use and Performance of Esophageal Outcome Measures in SSc

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/gastrointestinal-tract-involvement-in-juvenile-systemic-sclerosis-results-from-an-international-scoping-review-of-outcome-measures/