Short-Bowel Syndrome

The next (April 15th) issue of The Medical Letter will include an article on a new drug for a disorder called short-bowel syndrome, which is a consequence of not having enough small intestine to absorb necessary nutrients. The most common cause of this syndrome, which in its most severe form requires parenteral nutrition, seems to be multiple resections for Crohn’s disease. Crohn’s disease is fairly common, but short-bowel syndrome is rare because it only occurs after 70 or 80% of small bowel function is lost. Based on European surveys, the number of patients in the US might be between 5,000 and 10,000.

The new drug is teduglutide (Gattex is its brand name), an analog of an endogenous hormone produced by cells in the ileum and colon. This hormone, GLP-2, acts as a growth factor on the mucosa of the small bowel to increase the size of the villi and crypts that form the absorptive surface. Teduglutide is more effective than the native hormone in promoting growth of the mucosa because it has a longer half-life.

The ultimate purpose of increasing the size of villi and the depth of crypts is, of course, to increase the availability of nutrients, but from the patients’ practical perspective, the purpose is to decrease the need for parenteral nutrition. Short-bowel syndrome, rare as it is, is thought to be the most common reason for undertaking home infusion of parenteral fluids. These patients may require overnight infusions for 3-7 nights per week, at tremendous expense and inconvenience. If teduglutide can decrease the number of overnight infusions, or make it possible for some patients to be weaned off parenteral support altogether, it might justify the nearly $300,000 the drug will cost annually.

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