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.