Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
5719195 | The Journal of Pediatrics | 2017 | 15 Pages |
ObjectiveTo determine safety and pharmacodynamics/efficacy of teduglutide in children with intestinal failure associated with short bowel syndrome (SBS-IF).Study designThis 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 (0.0125âmg/kg/d [nâ=â8], 0.025âmg/kg/d [nâ=â14], 0.05âmg/kg/d [nâ=â15]) or received standard of care (SOC, nâ=â5). Descriptive summary statistics were used.ResultsAll patients experienced â¥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 â41% and â45%, respectively, with 0.025âmg/kg/d teduglutide and by â25% and â52% with 0.05âmg/kg/d teduglutide. In contrast, PN volume and calories changed by 0% and â6%, respectively, with 0.0125âmg/kg/d teduglutide and by 0% and â1% 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.ConclusionsTeduglutide was well tolerated in pediatric patients with SBS-IF. Teduglutide 0.025 or 0.05âmg/kg/d was associated with trends toward reductions in PN requirements and advancements in EN feeding in children with SBS-IF.Trial registrationClinicalTrials.gov: NCT01952080; EudraCT: 2013-004588-30.