Long‐term teduglutide associated with improved response in pediatric short bowel syndrome‐associated intestinal failure

Abstract Objectives Patients with short bowel syndrome‐associated intestinal failure (SBS‐IF) require long‐term parenteral nutrition and/or intravenous fluids (PN/IV) to maintain fluid or nutritional balance. We report the long‐term safety, efficacy, and predictors of response in pediatric patients with SBS‐IF receiving teduglutide over 96 weeks. Methods This was a pooled, post hoc analysis of two open‐label, long‐term extension (LTE) studies (NCT02949362 and NCT02954458) in children with SBS‐IF. Endpoints included treatment‐emergent adverse events (TEAEs) and clinical response (≥20% reduction in PN/IV volume from baseline). A multivariable linear regression identified predictors of teduglutide response; the dependent variable was mean change in PN/IV volume at each visit over 96 weeks. Results Overall, 85 patients were analyzed; 78 patients received teduglutide in the parent and/or LTE studies (any teduglutide [TED] group), while seven patients did not receive teduglutide in either the parent or LTE studies. Most TEAEs were moderate or severe in intensity in both groups. By week 96, 82.1% of patients from the any TED group achieved a clinical response, with a mean fluid decrease of 30.1 mL/kg/day and an energy decrease of 21.6 kcal/kg/day. Colon‐in‐continuity, non‐White race, older age at baseline, longer duration of teduglutide exposure, and increasing length of remaining small intestine were significantly associated with a reduction in mean PN/IV volume requirements. Conclusions In pediatric patients with SBS‐IF, teduglutide treatment resulted in long‐term reductions in PN/IV requirements. The degree of PN/IV volume reduction depended on the duration of teduglutide exposure, underlying bowel anatomy, and demographics.