Outcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome
dc.contributor.author | Carter, Beth A. | |
dc.contributor.author | Cohran, Valeria C. | |
dc.contributor.author | Cole, Conrad R. | |
dc.contributor.author | Corkins, Mark R. | |
dc.contributor.author | Dimmitt, Reed A. | |
dc.contributor.author | Duggan, Christopher | |
dc.contributor.author | Hill, Susan | |
dc.contributor.author | Horslen, Simon | |
dc.contributor.author | Lim, Joel D. | |
dc.contributor.author | Mercer, David F. | |
dc.contributor.author | Merritt, Russell J. | |
dc.contributor.author | Nichol, Peter F. | |
dc.contributor.author | Sigurdsson, Luther | |
dc.contributor.author | Teitelbaum, Daniel H. | |
dc.contributor.author | Thompson, John | |
dc.contributor.author | Vanderpool, Charles | |
dc.contributor.author | Vaughan, Juliana F. | |
dc.contributor.author | Li, Benjamin | |
dc.contributor.author | Youssef, Nader N. | |
dc.contributor.author | Venick, Robert S. | |
dc.contributor.author | Venick, Robert S. | |
dc.contributor.author | Kocoshis, Samuel A. | |
dc.contributor.department | Department of Pediatrics, School of Medicine | en_US |
dc.date.accessioned | 2017-09-15T14:39:20Z | |
dc.date.available | 2017-09-15T14:39:20Z | |
dc.date.issued | 2017-02 | |
dc.description.abstract | 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. | en_US |
dc.eprint.version | Final published version | en_US |
dc.identifier.citation | Carter, B. A., Cohran, V. C., Cole, C. R., Corkins, M. R., Dimmitt, R. A., Duggan, C., ... & Merritt, R. J. (2017). Outcomes from a 12-week, open-label, multicenter clinical trial of teduglutide in pediatric short bowel syndrome. The Journal of pediatrics, 181, 102-111. https://doi.org/10.1016/j.jpeds.2016.10.027 | en_US |
dc.identifier.uri | https://hdl.handle.net/1805/14086 | |
dc.language.iso | en | en_US |
dc.publisher | Elsevier | en_US |
dc.relation.isversionof | 10.1016/j.jpeds.2016.10.027 | en_US |
dc.relation.journal | The Journal of Pediatrics | en_US |
dc.rights | Attribution-NonCommercial-NoDerivs 3.0 United States | |
dc.rights.uri | https://creativecommons.org/licenses/by-nc-nd/3.0/us | |
dc.source | Publisher | en_US |
dc.subject | glucagon-like peptide 2 | en_US |
dc.subject | intestinal failure | en_US |
dc.subject | pediatric short bowel syndrome | en_US |
dc.title | Outcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome | en_US |
dc.type | Article | en_US |