Safety and long-term effectiveness of teduglutide in children with intestinal failure due to short bowel syndrome: a multicenter, post-marketing cohort study

BACKGROUND: Teduglutide, a glucagon-like peptide-2 (GLP-2) analog, was approved in the US for use in children with intestinal failure dependent on parenteral support (PS, including intravenous fluids and/or parenteral nutrition (PN)) in 2019. METHODS: Between May 2021 and December 2022, we enrolled 140 children with intestinal failure in a multi-center registry across 14 centers to evaluate the long-term (up to 3 years) safety and effectiveness of teduglutide. RESULTS: Among 120 participants in whom effectiveness was measured, the mean percent reduction in parenteral calories was 8.3% by 6 months, 16.5% by 12 months, 33.0% by 2 years and 49.5% by 3 years, estimated by linear mixed models. Cumulative incidence estimates for weaning from parenteral support (full enteral autonomy) were 7.5% by 6 months, 15.2% by 1 year, 26.4% by 2 years, and 32.7% by 3 years from baseline. Five patients had inflammatory/foveolar polyps. The modeled proportion of children with low weight-for-age Z score increased from 2.3% at baseline to 12.7% at 3 years (p < 0.01) and mean BMI Z-scores declined over follow-up as well. CONCLUSIONS: In children treated with teduglutide, we found a progressive and sustained reduction in parenteral support up to 3 years and recommend careful attention to the nutritional status of such patients. IMPACT: The GLP-2 analog medication teduglutide was approved by the FDA in 2019 for children receiving parenteral support for intestinal failure. We enrolled 140 such children to evaluate its safety and effectiveness profile in a post-marketing study. Substantial reductions in parenteral calories and volume were noted with a reasonable safety profile. Some reductions in nutritional status (weight and BMI but not height) were also noted. Careful monitoring of nutritional status while on this new medication is warranted. CLINICAL TRIAL REGISTRY SITE: NCT04832087.