Teduglutide for pediatric short bowel syndrome patients

ABSTRACT

Introduction: The goal for pediatric short bowel syndrome (SBS) patients is intestinal adaptation. Until recently, the medical management of pediatric SBS has centered on the prevention and treatment of complications in order to allow time for adaptation. Teduglutide, glucagon-like peptide 2 (GLP-2) analog, has recently been approved for use in pediatric SBS patients greater than 1 year of age as a novel agent to augment intestinal adaptation. Areas covered: This article reviews the pharmacology, safety, efficacy, and tolerability of GLP-2 analog teduglutide in pediatric patients greater than 1 year of age. We review all current studies and discuss teduglutide’s place in pediatric SBS therapy. Expert opinion: Teduglutide marks the first successful pharmacological intervention that augments the natural process of adaptation safely and effectively in SBS pediatric patients. More studies and further development are needed to optimize its potential in other pediatric patients.

KEYWORDS:

• Short bowel syndrome
• pediatrics
• glucagon-like peptide 2 analogue
• teduglutide
• revestive
• gattex
• glp-2 analogue

Article highlights

• Short bowel syndrome (SBS) is the primary cause of intestinal failure in pediatric patients with the goal of treatment being intestinal adaptation.

• Until recently, the medical management on SBS has centered on prevention and treatment of complications to provide time for intestinal adaptation.

• The introduction of GLP-2 analogue teduglutide marks the first successful pharmacological intervention that augments the natural process of adaptation with many reviews and studies showing safety and tolerability in adults.

• Reviewing the current literature and use in pediatrics, teduglutide has been shown to be effective, decreasing PN requirements and increasing enteral autonomy and intestinal growth.

• Teduglutide has also been shown to have no major safety concerns in pediatrics, but some adverse effects that may be confounded by underlying disease.

• Optimizing potential use of teduglutide includes assessing multiple factors including appropriate candidates and drug delivery form, timing, and administration and pediatric patients may benefit most from a specialized intestinal rehab program.

• There may be more potential for teduglutide in other patient populations and different formulations, but further translational research is needed.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties. Their institution and authors have participated in teduglutide trials with all grants paid to Seattle Children’s Research Institute, but authors have not received any personal funding or benefited from these trials in any way.

Reviewer disclosures

One reviewer would like to disclose that they have been involved as a researcher in the phase III study in adults to test the effect of Teduglutide on intestinal failure. The study has been finished more than 5 years ago. They also have received honoraria from Shire for scientific presentations in several symposia and that their institution has received research funding from Shire for basic science of intestinal failure and the effect of Teduglutide in mice. All other peer reviewers on this manuscript have no relevant financial or other relationships to disclose.