Predicting Teduglutide Treatment Response for Pediatric SBS-IF, With Valeria Cohran, MD

New research is shedding light on the characteristics of children with short bowel syndrome-associated intestinal failure (SBS-IF) by response to teduglutide, including attainment of consistent response and sustained enteral autonomy.1

The data were presented at the 2025 North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) Annual Meeting by Valeria Cohran, MD, a professor of pediatrics and the medical director of intestinal rehabilitation and transplant at Ann and Robert H Lurie Children's Hospital of Chicago.

Teduglutide, first approved by the US Food and Drug Administration for adults with SBS in 2012, was later granted approval for pediatric patients ≥ 1 year of age with SBS who need additional nutrition or fluids from intravenous feeding in 2019. The human glucagon-like peptide-2 analog reduces PN/IV dependency in children with SBS-IF, but identifying patient characteristics that predict response to teduglutide is crucial for guiding clinical decisions.1,2

“The reason we wanted to do this study was to evaluate the characteristics of children with short bowel syndrome and intestinal failure, and by looking at the response to teduglutide, trying to see what certain characteristics may predict who's going to be a responder to this medication,” Cohran told HCPLive.

This post hoc analysis pooled data from pediatric patients who took part in 1 of 2 open-label, multicenter, phase 3, long-term extension (LTE) studies over 96 weeks and their respective parent studies. Patients were grouped by whether or not they were consistent responders, defined as ≥20% PN/IV volume reduction from baseline for ≥90 consecutive days during parent and LTE studies, and whether or not they attained sustained enteral autonomy, defined as 100% reduction from baseline in PN/IV for ≥90 consecutive days.

In total, 23 patients were consistent responders and 46 were non-consistent responders. Of the non-consistent responders, 9 had no response, 1 had a response only during a parent study, 15 had a response only during an LTE study, and 21 had a response during both parent and LTE studies for <90 days. A total of 18 patients attained sustained enteral autonomy.

For patients with data available, consistent responders were significantly older (mean age, 7.9 vs 4.4 years; P <.001), had longer mean time since SBS diagnosis (7.1 vs 5.0 years; P = .016), and lower mean baseline PN/IV volume (53.5 vs 70.5 mL/kg/day; P = .030) and duration (10.7 vs 13.1 hours/day; P = .012) than non-consistent responders.

Investigators noted a significantly greater proportion of consistent responders had an ileocecal valve present (30.4% vs 15.2%; P = .047) and a lower proportion had some remaining colon than non-consistent responders (87.0% vs 100.0%; P = .012). Patients who attained sustained enteral autonomy were significantly older (mean age, 7.1 vs 5.0 years; P = .036), had greater mean length of remaining small intestine (57.3 vs 36.3 cm; P = .036), and lower mean baseline PN/IV volume (48.6 vs 70.6 mL/kg/day; P = .008) and duration (10.3 vs 12.7 hours/day; P = .025) than those who did not.

Univariate analyses identified older age at baseline and longer time since SBS diagnosis to be significantly associated with greater likelihood of consistent response, and older age at baseline and length of remaining small intestine to be significantly associated with greater likelihood of sustained enteral autonomy (P <.05). Of note, due to small sample sizes, multivariate analyses were not conducted.

“We need longer term studies, and as more patients are being treated with this drug, that's going to allow us to have more information about who is going to be a better responder to this agent,” Cohran said.

Editors’ Note: Cohran reports relevant disclosures with Takeda.

References

1. Cohran V, Carter B, Kocoshis S, et al. CHARACTERISTICS ASSOCIATED WITH RESPONSE TO TEDUGLUTIDE TREATMENT IN CHILDREN WITH SHORT BOWEL SYNDROME-ASSOCIATED INTESTINAL FAILURE: A POST HOC ANALYSIS OF LONG-TERM EXTENSION STUDIES. Presented at 2025 NASPGHAN Annual Meeting. November 5-9, 2025. Chicago, Illinois.

2. Pessoa Gingerich C. FDA Approves Teduglutide for Pediatric Use. HCPLive. May 17, 2019. Accessed December 10, 2025. https://www.hcplive.com/view/fda-approves-teduglutide-for-pediatric-use