Intestinal failure due to short bowel syndrome (SBS) is disabling as well as life-threatening in children. Teduglutide is a glucagon-like peptide-2 which promotes intestinal growth and bowel adaption. There is minimal data in children regarding the efficacy of this new medication, and the authors of this prospective, multi-center study followed 17 children with SBS who were treated with teduglutide at 0.05mg/kg/day via the subcutaneous route. All included patients had less than 100 cm of remaining bowel (except for 2 patients), were on parenteral nutrition (PN), and had no surgical intervention or changes in PN for 3 months prior to teduglutide use. At each clinic visit (baseline, 3 months, 6 months, and 12 months after therapy), information on PN volume, nutritional support, recorded stool losses, plasma citrulline levels, and the presence of adverse events were recorded. Any patient with a reduction in PN by 20% was defined as a “responder”. All patients were older than one year of age, and all patients developed intestinal failure after birth. The most common cause of intestinal failure was necrotizing enterocolitis. These patients were receiving an average 55 mL/kg/day of fluid volume daily (range 8-210 mL/kg/day) and were receiving 33 kcal/k/ day of nutritional support (range 0-65 kcal/kg/ day). Their mean initial citrulline level was 20 micromoles/L (range 7.8-51 micromoles/L).
A total of 15 of the 17 patients were able to complete one year of teduglutide. By the 3-month follow up, 3 patients had achieved full enteral autonomy. This trend continued with an additional 4 patients and then 3 patients reaching full enteral autonomy at 6 months and 12 months, respectively. Most patients were able to reduce their fluid volume and nutritional support, and in total, 14 of the 15 patients who finished the therapeutic study were responders to teduglutide. A 20% or greater reduction in PN support was noted in 47%, 87%, and 93% of patients at 3, 6, and 12 months respectively, while 17%, 44%, and 60% of patients were able to wean off of PN at 3, 6, and 12 months respectively. Stool output improved and citrulline levels increased in all patients throughout the study. The most common adverse events consisted of abdominal pain occurring in 30% of patients, followed by injection-site reactions, nausea, headaches, abdominal distention, and the presence of upper respiratory tract infections. Most of these side effects were mild or moderate.
This small study demonstrates promising results regarding the efficacy of teduglutide in the treatment of pediatric intestinal failure. More research is needed for children with an even greater loss of bowel as well as determination of cost savings associated with teduglutide use.
Boluda E, Ferreiro S, Moral O, Romero R, Terradillows I, Ramos R, Diaz M, Miquel B, Pinera I, Sanchez A, Sacristan R, Barea M, Villares J. Experience with teduglutide in pediatric short bowel syndrome: first real-life data. Journal of Pediatric Gastroenterology and Nutrition 2020; 71: 734-739.