J Pediatr Gastroenterol Nutr. 2022 Sep 1;75(3):293-298. doi: 10.1097/MPG.0000000000003541.Epub 2022 Aug 9.

Anat Guz-Mark ^1 2, Bayan Hino ³, Drora Berkowitz ⁴, Corina Hartman ⁵, Peri N Millman ⁶, Esther Orlanski-Meyer ⁷, Ron Shaoul ^8 9, Inna Spector-Cohen ⁸, Batia Weiss ^2 10, Tsili Zangen ¹¹, Raanan Shamir ^1 2

Author information

¹From the Institute of Gastroenterology Nutrition and Liver Diseases, Schneider Children's Medical Center of Israel, Petach-Tikva, Israel.

²the Sackler Faculty of Medicine, Tel-Aviv University, Tel-Aviv, Israel.

³the Department of Pediatrics, Ziv Medical Center, Azrieli Faculty of Medicine, Bar-ilan University, Safed, Israel.

⁴the Pediatric Gastroenterology Clinic, Bnai Zion Medical Center, Haifa, Israel.

⁵the Pediatric Gastroenterology and Nutrition Unit, Carmel Medical Center, Haifa, Israel.

⁶the Pediatric Gastrointestinal Clinic, Hadassah University Hospital, Jerusalem, Israel.

⁷the The Juliet Keidan Institute of Paediatric Gastroenterology and Nutrition, Shaare Zedek Medical Center, Jerusalem, Israel.

⁸the Pediatric Gastroenterology Institute, Ruth Rappaport Children's Hospital, Rambam Medical Center, Haifa, Israel.

⁹the Faculty of Medicine, Technion, Haifa, Israel.

¹⁰the Division of Pediatric Gastroenterology and Nutrition, Edmond & Lily Safra Children's Hospital, Sheba Medical Center, Tel-Hashomer, Israel.

¹¹the Pediatric Gastroenterology, Wolfson Medical Center, Holon, Israel.

Abstract

Objectives: The glucagon-like peptide-2 analog Teduglutide has been shown to enhance intestinal absorption and decrease parenteral nutrition (PN) requirements in short bowel syndrome (SBS). As data in children is limited, we evaluated nationwide real-life experience and treatment outcome in children with SBS.

Methods: Longitudinal data of children treated with Teduglutide for ≥3 months was collected. Data included demographic and medical background, anthropometrics, laboratory assessments and PN requirements. Treatment response was defined as >20% reduction in PN requirement.

Results: The study included 13 patients [54% males, median (interquartile range {IQR}) age of 6 (4.7-7) years]. The most common SBS etiology was necrotizing enterocolitis (38%), and median (IQR) small bowel length was 20 (15-40) cm. Teduglutide treatment ranged between 3 and 51 months [median (IQR) of 18 (12-30) months], with 10 patients (77%) treated >1 year. Response to treatment was observed in 8 patients (62%), with a mean [±standard deviation (SD)] treatment duration of 5.9 (±3.2) months. Among responders, 2 patients were weaned off PN and additional 4 decreased PN needs by >40%. There was a median (IQR) reduction in PN volume/kg of 36% (15%-55%) and in PN energy/kg of 27% (6%-58%). Response was not associated with patients' background, and no correlation was found with bowel length or PN dependency at baseline.

Conclusions: Real-life response to Teduglutide is highly variable among children with SBS. While most patients did reach 20% reduction in PN, less achieved further significant reduction or enteral autonomy. No predictive factors of response to treatment were identified, and large multicenter studies are needed to elucidate predictive factors and long-term outcome.