Abstract

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

 Pediatr Gastroenterol Nutr. 2024 Jun 14. doi: 10.1002/jpn3.12276. Online ahead of print.

 

Paul W Wales 1Susan Hill 2Ian Robinson 3Bram P Raphael 3Cheney Matthews 3Valeria Cohran 4Beth Carter 5Robert Venick 6Samuel Kocoshis 7

 
     

Author information

1Division of Pediatric General and Thoracic Surgery, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA.

2Department of Paediatric Gastroenterology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.

3Takeda Pharmaceuticals, Inc., Lexington, Massachusetts, USA.

4Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.

5Children's Hospital Los Angeles and Keck School of Medicine, University of Southern California, Los Angeles, California, USA.

6Division of Gastroenterology, Hepatology, and Nutrition, UCLA Mattel Children's Hospital, Los Angeles, California, USA.

7Division of Pediatric Surgery, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA.

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.

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