Outcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome - 18/04/17
, Valeria C. Cohran, MD, MS 2, Conrad R. Cole, MD, MPH, MSc 3, Mark R. Corkins, MD 4, Reed A. Dimmitt, MD, MSPH 5, Christopher Duggan, MD, MPH 6, Susan Hill, MRCPCH, DM 7, Simon Horslen, MB, ChB, FRCPCH 8, Joel D. Lim, MD 9, David F. Mercer, MD, PhD, FRCS(C) 10, Russell J. Merritt, MD, PhD 11, Peter F. Nichol, MD, PhD 12, Luther Sigurdsson, MD 13, Daniel H. Teitelbaum, MD 14, *, John Thompson, MD 15, Charles Vanderpool, MD 16, Juliana F. Vaughan, MD 17, Benjamin Li, MS 18, †, Nader N. Youssef, MD 19, ‡, Robert S. Venick, MD 20, Samuel A. Kocoshis, MD 3Abstract |
Objective |
To determine safety and pharmacodynamics/efficacy of teduglutide in children with intestinal failure associated with short bowel syndrome (SBS-IF).
Study design |
This 12-week, open-label study enrolled patients aged 1-17 years with SBS-IF who required parenteral nutrition (PN) and showed minimal or no advance in enteral nutrition (EN) feeds. Patients enrolled sequentially into 3 teduglutide cohorts (0.0125 mg/kg/d [n = 8], 0.025 mg/kg/d [n = 14], 0.05 mg/kg/d [n = 15]) or received standard of care (SOC, n = 5). Descriptive summary statistics were used.
Results |
All patients experienced ≥1 treatment-emergent adverse event; most were mild or moderate. No serious teduglutide-related treatment-emergent adverse events occurred. Between baseline and week 12, prescribed PN volume and calories (kcal/kg/d) changed by a median of −41% and −45%, respectively, with 0.025 mg/kg/d teduglutide and by −25% and −52% with 0.05 mg/kg/d teduglutide. In contrast, PN volume and calories changed by 0% and −6%, respectively, with 0.0125 mg/kg/d teduglutide and by 0% and −1% with SOC. Per patient diary data, EN volume increased by a median of 22%, 32%, and 40% in the 0.0125, 0.025, and 0.05 mg/kg/d cohorts, respectively, and by 11% with SOC. Four patients achieved independence from PN, 3 in the 0.05 mg/kg/d cohort and 1 in the 0.025 mg/kg/d cohort. Study limitations included its short-term, open-label design, and small sample size.
Conclusions |
Teduglutide was well tolerated in pediatric patients with SBS-IF. Teduglutide 0.025 or 0.05 mg/kg/d was associated with trends toward reductions in PN requirements and advancements in EN feeding in children with SBS-IF.
Trial registration |
ClinicalTrials.gov: NCT01952080; EudraCT: 2013-004588-30.
Il testo completo di questo articolo è disponibile in PDF.Keywords : glucagon-like peptide 2, intestinal failure, pediatric short bowel syndrome, short gut syndrome
Abbreviations : AE, EN, GI, GLP, IF, ITT, PD, PN, SBS, SBS-IF, SOC, TEAE, TESAE
Mappa
| Funded by NPS Pharmaceuticals, Inc., Lexington, MA, a wholly owned indirect subsidiary of Shire. Potential conflicts of interest are available at www.jpeds.com (Appendix). |
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| Portions of the study were presented as poster or oral presentations at: European Society for Clinical Nutrition and Metabolism, September 5-8, 2015, Lisbon, Portugal; North American Society for Pediatric Gastroenterology, Hepatology and Nutrition, October 7-11, 2015, Washington, DC; American Society for Parenteral and Enteral Nutrition Clinical Nutrition Week, January 16-19, 2016, Austin, TX; British Society of Paediatric Gastroenterology, Hepatology and Nutrition, January 27-29, 2016, Bristol, United Kingdom; Sociedad Española de Gastroenterología, Hepatología y Nutrición Pediátrica, May 12-14, 2016, Gijón, Spain; and European Society for Paediatric Gastroenterology, Hepatology and Nutrition, May 25-28, 2016, Athens, Greece. |
Vol 181
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