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JPEN J Parenter Enteral Nutr. 2019 Sep 8. doi: 10.1002/jpen.1690. [Epub ahead of print]

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

Author information

1
Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
2
Children's Hospital Los Angeles and Keck School of Medicine, University of Southern California, Los Angeles, California, USA.
3
Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
4
Birmingham Children's Hospital NHS Foundation Trust, Birmingham, UK.
5
Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.
6
Seattle Children's Hospital and University of Washington School of Medicine, Seattle, Washington, USA.
7
Shire Human Genetic Therapies, Inc, Lexington, Massachusetts, USA.
8
MedStar Georgetown University Hospital, Washington, District of Columbia, USA.
9
University of Nebraska Medical Center, Omaha, Nebraska, USA.
10
Children's Hospital, Pediatric Research Center, University of Helsinki, Helsinki University Hospital, Helsinki, Finland.
11
Mattel Children's Hospital UCLA, Department of Pediatrics, Los Angeles, California, USA.
12
The Hospital for Sick Children, Toronto, Ontario, Canada.
13
Shire Human Genetic Therapies, Inc, Cambridge, Massachusetts, USA.

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.

KEYWORDS:

gastroenterology; parenteral nutrition; pediatrics; short bowel syndrome

PMID:
31495952
DOI:
10.1002/jpen.1690

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