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Safety and Efficacy of Teduglutide in Pediatric Patients With Intestinal Failure due to Short Bowel Syndrome: A 24‐Week, Phase III Study
Author(s) -
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.
Publication year - 2020
Publication title -
journal of parenteral and enteral nutrition
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.935
H-Index - 98
eISSN - 1941-2444
pISSN - 0148-6071
DOI - 10.1002/jpen.1690
Subject(s) - medicine , adverse effect , clinical endpoint , parenteral nutrition , short bowel syndrome , gastroenterology , vomiting , nausea , enteral administration , randomized controlled trial
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.