Nutrition Week (Day 4) Trophic Hormone for Pediatric Short Bowel Syndrome

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A recent study (BA Carter et al. J Pediatr 2017; 181: 102-11) provides some preliminary data on the use of glucagon-like peptide-2 (GLP-2) (Teduglutide) for pediatric short bowel syndrome (SBS).

This was a 12-week, open-label study in patients aged 1-17 years with intestinal failure (IF) associated with SBS. Prior to the study, patients had shown little to minimal advance in enteral nutrition for at least 3 months. Three doses of GLP-2 (0.0125 mg/kg/d, 0.025 mg/kg/day, and 0.05 mg/kg/day).

Key findings:

  • All treated patients (37) experienced mild or moderate adverse effects, including vomiting, pyrexia, catheter-related complications, and upper respiratory tract infections. No serious adverse events were identified. In the 5 patients who received standard care, adverse effects were recorded as well, including upper respiratory tract infections in 2 (40%) which was similar to the other groups.
  • By week 12, parenteral nutrition (PN) volume and calories were reduced in the higher dosed groups.  In the 0.025 mg/kg/day group, PN volume dropped by 41% and calories by 45%.  In the 0.05 mg/kg/day group, PN volume dropped by 25% and calories by 52%. Virtually no change in these parameters occurred in the lowest dose (0.0125 mg/kg/day) with no change in volume and 6% drop in calories.
  • Enteral feeding volume occurred in all groups: 22%, 32%, and 40% in the groups and was directly related to the GLP-2 dosing.
  • Citrulline levels (a biomarker of enteral autonomy) were monitored “but the results were clouded by wide variability of baseline values.”  In adult studies, citrulline levels increased significantly.

My take: This open-label study has many limitations; further studies are planned (ClinicalTrials.gov, NCT02682381). Nevertheless, this study indicates that GLP-2 holds promise as a therapy for SBS/IF.

Another slide in a recent lecture on PNALD (slide derived from Conrad Cole lecture in Octobler 2015 -available at Pediatric Nutritionist Blog, slide 49):

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