Outcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome

dc.contributor.authorCarter, Beth A.
dc.contributor.authorCohran, Valeria C.
dc.contributor.authorCole, Conrad R.
dc.contributor.authorCorkins, Mark R.
dc.contributor.authorDimmitt, Reed A.
dc.contributor.authorDuggan, Christopher
dc.contributor.authorHill, Susan
dc.contributor.authorHorslen, Simon
dc.contributor.authorLim, Joel D.
dc.contributor.authorMercer, David F.
dc.contributor.authorMerritt, Russell J.
dc.contributor.authorNichol, Peter F.
dc.contributor.authorSigurdsson, Luther
dc.contributor.authorTeitelbaum, Daniel H.
dc.contributor.authorThompson, John
dc.contributor.authorVanderpool, Charles
dc.contributor.authorVaughan, Juliana F.
dc.contributor.authorLi, Benjamin
dc.contributor.authorYoussef, Nader N.
dc.contributor.authorVenick, Robert S.
dc.contributor.authorVenick, Robert S.
dc.contributor.authorKocoshis, Samuel A.
dc.contributor.departmentDepartment of Pediatrics, School of Medicineen_US
dc.date.accessioned2017-09-15T14:39:20Z
dc.date.available2017-09-15T14:39:20Z
dc.date.issued2017-02
dc.description.abstractObjective 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.versionFinal published versionen_US
dc.identifier.citationCarter, 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.2016.10.027en_US
dc.identifier.urihttps://hdl.handle.net/1805/14086
dc.language.isoenen_US
dc.publisherElsevieren_US
dc.relation.isversionof10.1016/j.jpeds.2016.10.027en_US
dc.relation.journalThe Journal of Pediatricsen_US
dc.rightsAttribution-NonCommercial-NoDerivs 3.0 United States
dc.rights.urihttps://creativecommons.org/licenses/by-nc-nd/3.0/us
dc.sourcePublisheren_US
dc.subjectglucagon-like peptide 2en_US
dc.subjectintestinal failureen_US
dc.subjectpediatric short bowel syndromeen_US
dc.titleOutcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndromeen_US
dc.typeArticleen_US
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