Safety and efficacy of teduglutide after 52 weeks of treatment in patients with short bowel intestinal failure.

Abstract

BACKGROUND & AIMS:

Although home parenteral nutrition (PN) can save the lives of patients with massive bowel loss that results in short-bowel syndrome and intestinal failure, quality of life is impaired by PN and its complications. We examined the 12-month tolerability and efficacy of teduglutide to reduce PN dependency.

METHODS:

Patients who received teduglutide (0.05 or 0.10 mg/kg/d) for 24 weeks in a randomized controlled trial were eligible for a 28-week double-blind extension study; 52 patients were given 52 weeks of the same doses of teduglutide. We investigated the safety, tolerability, and clinical efficacy (defined as a clinically meaningful ≥20% reduction in weekly PN volume from baseline) at week 52.

RESULTS:

The most common adverse events reported included headache (35%), nausea (31%), and abdominal pain (25%); 7 patients withdrew because of adverse events (gastrointestinal disorders in 4). Both groups had progressive reduction in PN. At week 52, 68% of the 0.05-mg/kg/d and 52% of the 0.10-mg/kg/d dose group had a ≥20% reduction in PN, with a reduction of 1 or more days of PN dependency in 68% and 37%, respectively. Four patients achieved complete independence from PN.

CONCLUSIONS:

For patients with short-bowel syndrome intestinal failure, the efficacy of teduglutide was maintained over 52 weeks and the safety profile was sufficient for it to be considered for long-term use. Further studies are needed to determine whether these effects will translate into improved quality of life and reduced PN complications. ClinicalTrials.gov number, NCT00172185.

• Authors: Stephen J D O'Keefe, Palle B Jeppesen, Richard Gilroy, Marek Pertkiewicz, Johane P Allard, Bernard Messing
• Journal: Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association (Clin Gastroenterol Hepatol) Vol. 11 Issue 7 Pg. 815-23.e1-3 (Jul 2013) ISSN: 1542-7714 [Electronic] United States
• PMID: 23333663 (Publication Type: Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2013 AGA Institute. Published by Elsevier Inc. All rights reserved.

Chemical References

• Gastrointestinal Agents
• Peptides
• teduglutide

Topics

• Adult
• Aged
• Aged, 80 and over
• Double-Blind Method
• Female
• Gastrointestinal Agents (adverse effects, therapeutic use)
• Humans
• Male
• Middle Aged
• Parenteral Nutrition
• Peptides (adverse effects, therapeutic use)
• Short Bowel Syndrome (drug therapy)
• Treatment Outcome
• Young Adult