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

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.

Staple line ulcers: a cause of chronic GI bleeding following STEP procedure.

Intestinal failure (IF) results from a critical reduction in the functional intestinal mass resulting in dependence on total parenteral nutrition (TPN) for growth and survival. Short bowel syndrome (SBS) is the most common cause of intestinal failure in pediatrics. Following resection, the small bowel undergoes adaptation, a process wherein the bowel elongates and dilates in order to increase intestinal absorptive capacity. Small bowel dilatation can lead to dysmotility and small bowel bacterial overgrowth which may further enhance feeding intolerance. Bowel lengthening procedures are beneficial when there is significant dilatation of the small bowel and subsequent inability to advance enteral feeds. We describe a patient with intestine failure and short bowel syndrome due to gastroschisis who, presented with anemia and occult gastrointestinal bleeding, following Serial Transverse Enteroplasty procedure (STEP). Video capsule endoscopy (VCE) revealed multiple ulcerations at surgical staple sites throughout the distal 2/3 of the remaining small intestine which were the likely source of intestinal blood loss.