title: Outcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome.
abstract: To determine safety and pharmacodynamics/efficacy of teduglutide in children with intestinal failure associated with short bowel syndrome (SBS-IF).
  
  
  
  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.
  
  
  
  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.
  
  
  
  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.
  
  
  
  ClinicalTrials.gov:NCT01952080; EudraCT: 2013-004588-30.
authors.affiliation: Department of Pediatric Medicine, Gastroenterology, Hepatology and Nutrition, Texas Children's Hospital and Baylor College of Medicine, Houston, TX. Electronic address: bac@bcm.edu.
authors.forename: Beth A
authors.initials: BA
authors.lastname: Carter
authors.affiliation: Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL.
authors.forename: Valeria C
authors.initials: VC
authors.lastname: Cohran
authors.affiliation: Division of Gastroenterology, Hepatology and Nutrition, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
authors.forename: Conrad R
authors.initials: CR
authors.lastname: Cole
authors.affiliation: Pediatric Gastroenterology, LeBonheur Children's Hospital and University of Tennessee Health Science Center, Memphis, TN.
authors.forename: Mark R
authors.initials: MR
authors.lastname: Corkins
authors.affiliation: Division of Pediatric Gastroenterology, Hepatology and Nutrition, University of Alabama at Birmingham and Children's of Alabama, Birmingham, AL.
authors.forename: Reed A
authors.initials: RA
authors.lastname: Dimmitt
authors.affiliation: Division of Gastroenterology, Hepatology and Nutrition, Boston Children's Hospital, Boston, MA.
authors.forename: Christopher
authors.initials: C
authors.lastname: Duggan
authors.affiliation: Gastroenterology Department, Great Ormond Street Hospital for Children, London, United Kingdom.
authors.forename: Susan
authors.initials: S
authors.lastname: Hill
authors.affiliation: Hepatobiliary and Intestinal Failure Programs, Seattle Children's Hospital, Seattle, WA.
authors.forename: Simon
authors.initials: S
authors.lastname: Horslen
authors.affiliation: Intestinal Rehabilitation Center, Children's Mercy Hospitals & Clinics, Kansas City, MO.
authors.forename: Joel D
authors.initials: JD
authors.lastname: Lim
authors.affiliation: Intestinal Rehabilitation Program, Nebraska Medical Center, Omaha, NE.
authors.forename: David F
authors.initials: DF
authors.lastname: Mercer
authors.affiliation: Gastroenterology, Hepatology and Nutrition, Children's Hospital Los Angeles and Keck School of Medicine, University of Southern California, Los Angeles, CA.
authors.forename: Russell J
authors.initials: RJ
authors.lastname: Merritt
authors.affiliation: Department of Surgery, University of Wisconsin School of Medicine and Public Health, Madison, WI.
authors.forename: Peter F
authors.initials: PF
authors.lastname: Nichol
authors.affiliation: Division of Pediatric Gastroenterology, University of Wisconsin School of Medicine and Public Health, Madison, WI.
authors.forename: Luther
authors.initials: L
authors.lastname: Sigurdsson
authors.affiliation: Pediatric Surgery, CS Mott Children's Hospital, Ann Arbor, MI.
authors.forename: Daniel H
authors.initials: DH
authors.lastname: Teitelbaum
authors.affiliation: Division of Pediatric Gastroenterology and Nutrition, Children's Hospital at Montefiore, New York, NY.
authors.forename: John
authors.initials: J
authors.lastname: Thompson
authors.affiliation: Division of Pediatric Gastroenterology, Hepatology & Nutrition, Riley Hospital for Children and Indiana University School of Medicine, Indianapolis, IN.
authors.forename: Charles
authors.initials: C
authors.lastname: Vanderpool
authors.affiliation: Gastroenterology, Nutrition, and Hepatology, Arkansas Children's Hospital, Little Rock, AR.
authors.forename: Juliana F
authors.initials: JF
authors.lastname: Vaughan
authors.affiliation: Department of Statistics, NPS Pharmaceuticals, Inc., Lexington, MA.
authors.forename: Benjamin
authors.initials: B
authors.lastname: Li
authors.affiliation: Department of Clinical Research and Development, NPS Pharmaceuticals, Inc., Lexington, MA.
authors.forename: Nader N
authors.initials: NN
authors.lastname: Youssef
authors.affiliation: Departments of Pediatrics and Surgery, Mattel Children's Hospital UCLA, Los Angeles, CA.
authors.forename: Robert S
authors.initials: RS
authors.lastname: Venick
authors.affiliation: Division of Gastroenterology, Hepatology and Nutrition, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
authors.forename: Samuel A
authors.initials: SA
authors.lastname: Kocoshis
mesh_terms: D000293:Adolescent; D000367:Age Factors; D002648:Child; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D004750:Enteral Nutrition / Q000379:methods*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D061214:Patient Safety; D010455:Peptides / Q000008:administration & dosage* / Q000009:adverse effects; D011446:Prospective Studies; D018570:Risk Assessment; D012720:Severity of Illness Index; D012737:Sex Factors; D012778:Short Bowel Syndrome / Q000175:diagnosis / Q000188:drug therapy* / Q000628:therapy; D016896:Treatment Outcome
chemical_list: D010455:Peptides; C494910:teduglutide
keywords: glucagon-like peptide 2; intestinal failure; pediatric short bowel syndrome; short gut syndrome