@article{40539398be344a5d8dce5ebd65e0f348,
title = "Safety and Efficacy of Teduglutide in Pediatric Patients With Intestinal Failure due to Short Bowel Syndrome: A 24-Week, Phase III Study",
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",
author = "Kocoshis, {Samuel A.} and Merritt, {Russell J.} and Susan Hill and Susan Protheroe and Carter, {Beth A.} and Simon Horslen and Simin Hu and Kaufman, {Stuart S.} and Mercer, {David F.} and Pakarinen, {Mikko P.} and Venick, {Robert S.} and Wales, {Paul W.} and Grimm, {Andrew A.}",
note = "Funding Information: Under the direction of the authors, editorial support was provided by Alan Storey, PhD, and Sheila M. Curristin, PhD, of Complete Healthcare Communications, LLC, a CHC Group company (North Wales, PA, USA). We acknowledge the other clinical investigators who treated patients and collected data for this study: Drs Andreas Busch (University Children's Hospital, T{\"u}bingen, Germany), Valeria Cohran (Ann & Robert H. Lurie Children's Hospital, Chicago, IL, USA), Antonella Diamanti (Ospedale Pediatrico Bambino Ges{\`u}, Rome, Italy), Dominique Hermans (Cliniques Universitaires Saint-Luc, Brussels, Belgium), Mercedes Martinez (Morgan Stanley Children's Hospital GI Nutrition, New York, NY, USA), Peter Nichol (University of Wisconsin School of Medicine and Public Health, Madison, WI, USA), Sue Rhee (UCSF Benioff Children's Hospital, San Francisco, CA, USA), Debra Sudan (Duke Medical Center, Durham, NC, USA), Natalie Terry (University of Pennsylvania Medical Center, Philadelphia, PA, USA), John Thompson (Montefiore Medical Center Child Spc, New York, NY, USA), Rabindranath Persad (Walter C. Mackenzie Health Science Centre, Edmonton, AB, Canada), Bram Raphael (Boston Children's Hospital, Boston, MA, USA), and Charles Vanderpool (Riley Hospital for Children, Indianapolis, IN, USA). We thank the patients and their families who participated in this study. We thank the network of clinical coordinators at the participating study centers. We thank Yaron Avitzur (The Hospital for Sick Children, Toronto, Canada), Jutta Koeglmeier and Sophie Foxall (Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK), Laura Merras-Salmio (Children's Hospital, Pediatric Research Center, University of Helsinki, Helsinki University Hospital, Helsinki, Finland), and George Yanni and Kim Rinauro (Children's Hospital Los Angeles and Keck School of Medicine, University of Southern California, Los Angeles, CA, USA). Publisher Copyright: {\textcopyright} 2019 The Authors. Journal of Parenteral and Enteral Nutrition published by Wiley Periodicals, Inc. on behalf of American Society for Parenteral and Enteral Nutrition.",
year = "2020",
month = may,
day = "1",
doi = "10.1002/jpen.1690",
language = "English (US)",
volume = "44",
pages = "621--631",
journal = "Journal of Parenteral and Enteral Nutrition",
issn = "0148-6071",
publisher = "John Wiley & Sons Inc.",
number = "4",
}