Safety and Efficacy of Teduglutide in Pediatric Patients With Intestinal Failure due to Short Bowel Syndrome : A 24-Week, Phase III Study

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Kocoshis , S A , Merritt , R J , Hill , S , Protheroe , S , Carter , B A , Horslen , S , Hu , S , Kaufman , S S , Mercer , D F , Pakarinen , M P , Venick , R S , Wales , P W & Grimm , A A 2020 , ' Safety and Efficacy of Teduglutide in Pediatric Patients With Intestinal Failure due to Short Bowel Syndrome : A 24-Week, Phase III Study ' , Journal of Parenteral and Enteral Nutrition , vol. 44 , no. 4 , pp. 621-631 . https://doi.org/10.1002/jpen.1690

Title: Safety and Efficacy of Teduglutide in Pediatric Patients With Intestinal Failure due to Short Bowel Syndrome : A 24-Week, Phase III Study
Author: Kocoshis, Samuel A.; Merritt, Russell J.; Hill, Susan; Protheroe, Susan; Carter, Beth A.; Horslen, Simon; Hu, Simin; Kaufman, Stuart S.; Mercer, David F.; Pakarinen, Mikko P.; Venick, Robert S.; Wales, Paul W.; Grimm, Andrew A.
Contributor: University of Helsinki, Children's Hospital
Date: 2020-05
Language: eng
Number of pages: 11
Belongs to series: Journal of Parenteral and Enteral Nutrition
ISSN: 0148-6071
URI: http://hdl.handle.net/10138/315802
Abstract: Background This study evaluated the safety and efficacy of teduglutide in pediatric patients with short bowel syndrome-associated intestinal failure (SBS-IF). Methods A 24-week, phase III trial with 2 randomized, double-blind teduglutide dose groups and a nonblinded standard of care (SOC) arm was used; patients received 0.025 mg/kg or 0.05 mg/kg teduglutide once daily. Safety end points included treatment-emergent adverse events (TEAEs) and growth parameters. The primary efficacy/pharmacodynamic end point was the number of patients who achieved a >= 20% reduction in parenteral support (PS) from baseline at week 24. Results All 59 enrolled patients completed the study (0.025 mg/kg, n = 24; 0.05 mg/kg, n = 26; SOC, n = 9). Baseline demographics and disease characteristics were comparable among groups. TEAEs were reported by 98% and 100% of patients in the teduglutide and SOC groups, respectively. The most common TEAEs in the teduglutide-treated groups were pyrexia and vomiting. The primary end point was achieved by 13 (54.2%), 18 (69.2%), and 1 (11.1%) patients who received 0.025 mg/kg teduglutide, 0.05 mg/kg teduglutide, and SOC, respectively (P <0.05 vs SOC). Both 0.025-mg/kg and 0.05-mg/kg teduglutide groups showed clinically significant reductions in PS volume (P <0.05 vs SOC), PS calories, days per week and hours per day of PS infusions, and increases in enteral nutrition and plasma citrulline at week 24 compared with baseline. Two (8.3%, 0.025 mg/kg teduglutide) and 3 patients (11.5%, 0.05 mg/kg teduglutide) achieved enteral autonomy. Conclusion The safety profile of teduglutide was similar to that reported previously in children and adults. Treatment with teduglutide was associated with significant reductions in PS for pediatric patients with SBS-IF over 24 weeks.
Subject: gastroenterology
parenteral nutrition
pediatrics
short bowel syndrome
ADAPTATION
CHILDREN
3121 General medicine, internal medicine and other clinical medicine
3143 Nutrition
3123 Gynaecology and paediatrics
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