Eur J Pediatr. 2024 Aug;183(8):3173-3182.doi: 10.1007/s00431-024-05577-5. Epub 2024 Apr 26.

Marta Germán-Díaz ¹, Alida Alcolea ², Vanessa Cabello ³, Javier Blasco-Alonso ⁴, Alejandro Rodríguez ⁵, Rafael Galera ⁶, Ruth García-Romero ⁷, Carmen Romero ⁸, Rocío González-Sacristán ², Susana Redecillas-Ferreiro ³, José Manuel Moreno-Villares ⁹, Esther Ramos-Boluda ²

Author information

¹Pediatric Gastroenterology, Hepatology and Nutrition Department, Hospital Universitario, 12 de Octubre, Madrid, Spain. marta.german@salud.madrid.org.

²Intestinal Rehabilitation Unit, Hospital La Paz, Madrid, Spain.

³Pediatric Gastroenterology and Nutrition Support Unit, Hospital Vall D'Hebrón, Barcelona, Spain.

⁴Pediatric Gastroenterology and Nutrition Department, Hospital Regional Universitario de Málaga, Málaga, Spain.

⁵Pediatric Gastroenterology Department, Hospital Universitario Virgen del Rocío, Seville, Spain.

⁶Pediatric Gastroenterology and Nutrition Department, Hospital Universitario Torrecárdenas, Almería, Spain.

⁷Pediatric Gastroenterology and Nutrition Department, Hospital Universitario Miguel Servet, Zaragoza, Spain.

⁸Research Institute, Hospital Universitario, 12 de Octubre, Madrid, Spain.

⁹Pediatric Department, Clínica Universitaria de Navarra, Madrid, Spain.

Abstract

Teduglutide is a glucagon-like-peptide-2 analogue that reduces the need for parenteral support in patients with short bowel syndrome (SBS). Nevertheless, data about long-term therapy with teduglutide in children are still scarce. Our objective was to describe the real-life experience with teduglutide in children with SBS over the last 5 years in Spain. This was a national multicentre and prospective study of paediatric patients with intestinal failure (IF) treated with teduglutide for at least 3 months. The data included demographic characteristics, medical background, anthropometric data, laboratory assessments, adverse events, and parenteral nutrition (PN) requirements. Treatment response was defined as a > 20% reduction in the PN requirement. The data were collected from the Research Electronic Data Capture (REDCap) database. Thirty-one patients from seven centres were included; the median age at the beginning of the treatment was 2.3 (interquartile range (IQR) 1.4-4.4) years; and 65% of the patients were males. The most frequent cause of IF was SBS (94%). The most common cause of SBS was necrotizing enterocolitis (35%). The median residual bowel length was 29 (IQR 12-40) cm. The median duration of teduglutide therapy was 19 (IQR 12-36) months, with 23 patients (74%) treated for > 1 year and 9 treated for > 3 years. The response to treatment was analysed in 30 patients. Twenty-four patients (80%) had a reduction in their weekly PN energy > 20% and 23 patients (77%) had a reduction in their weekly PN volume > 20%. Among the responders, 9 patients (29%) were weaned off PN, with a median treatment duration of 6 (IQR 4.5-22) months. The only statistically significant finding demonstrated an association between a > 20% reduction in the weekly PN volume and a younger age at the start of treatment (p = 0.028). Conclusions: Teduglutide seems to be an effective and safe treatment for paediatric patients with IF. Some patients require a prolonged duration of treatment to achieve enteral autonomy. Starting treatment with teduglutide at a young age is associated with a higher response rate. What is Known: • Glucagon-like peptide-2 (GLP-2) plays a crucial role in the regulation of intestinal adaptation in short bowel syndrome (SBS). Teduglutide is a GLP-2 analog that reduces the need for parenteral support in patients with SBS. • Data about long-term therapy with teduglutide in children in real life are still scarce. What is New: • Most pediatric patients with SBS respond in a satisfactory manner to teduglutide treatment. The occurrence of long-term adverse effects is exceptional. • Starting treatment with the drug at a young age is associated with a greater response rate.