J Pediatr Gastroenterol Nutr. 2023 Sep 1;77(3):339-346.doi: 10.1097/MPG.0000000000003867. Epub 2023 Jun 26.

Masahiro Chiba ¹, Kouji Masumoto ², Tatsuru Kaji ³, Toshiharu Matsuura ⁴, Mayako Morii ⁵, Andrew Fagbemi ⁶, Susan Hill ⁷, Mikko P Pakarinen ⁸, Susan Protheroe ⁹, Arun Urs ¹⁰, Szu-Ta Chen ¹¹, Sho Sakui ¹², Eri Udagawa ¹³, Motoshi Wada ^14 15

Author information

¹From the Division of Pediatric Surgery, Department of Surgery, Showa University, Tokyo, Japan.

²the Department of Pediatric Surgery, Faculty of Medicine, University of Tsukuba, Tsukuba, Japan.

³the Department of Pediatric Surgery, Research Field in Medicine and Health Sciences, Medical and Dental Sciences Area, Research and Education Assembly, Kagoshima University, Kagoshima, Japan.

⁴the Department of Pediatric Surgery, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan.

⁵the Department of Pediatric Surgery, Akita University Graduate School of Medicine, Akita, Japan.

⁶the Department of Paediatric Gastroenterology, Royal Manchester Children's Hospital, Manchester, UK.

⁷the Department of Paediatric Gastroenterology, Great Ormond Street Hospital for Children, NHS Foundation Trust and UCL Institute of Child Health, London, UK.

⁸the Pediatric Research Center, Children's Hospital, University of Helsinki, Helsinki University Hospital, Helsinki, Finland.

⁹the Department of Gastroenterology and Nutrition, Birmingham Women and Children's Hospital, Birmingham, UK.

¹⁰the Department of Paediatric Gastroenterology, Sheffield Children's Hospital, Sheffield, UK.

¹¹Takeda Development Center Americas, Inc., Boston, MA, USA.

¹²Statistical and Quantitative Sciences, Data Sciences Institute, Takeda Pharmaceutical Company Limited, Osaka, Japan.

¹³Japan Medical Office, Takeda Pharmaceutical Company Limited, Tokyo, Japan.

¹⁴the Department of Pediatric Surgery, Tohoku University Graduate School of Medicine, Sendai, Japan.

¹⁵the Division of Pediatric Surgery, Department of Surgery, Tohoku University Hospital, Sendai, Japan.

Abstract

Objectives: Our objective was to evaluate the short- and long-term safety and efficacy of teduglutide treatment in infants and children with short bowel syndrome with intestinal failure (SBS-IF).

Methods: Two open-label phase 3 studies and 1 extension study investigated the short- and long-term safety and efficacy of teduglutide (0.05 mg/kg/day) in infants and children with SBS-IF: NCT03571516, 24-week study of infants who were randomized to receive teduglutide or standard of care (SoC); NCT02980666, 24-week study of infants and children who all received teduglutide; and NCT03268811, 24-week extension study of patients who completed NCT02980666 (patients could receive up to 48 weeks of total treatment).

Results: Twelve infants and 8 children enrolled in the core studies, and 2 infants and 7 children in the extension study. After 24 weeks of treatment, parenteral support (PS) requirements reduced by ≥20% from baseline for 4 infants (57.1%) and 4 children (66.7%) receiving teduglutide and for 2 infants receiving SoC (50.0%). One infant (50.0%) and 4 children (80.0%) receiving teduglutide maintained the ≥20% reduction in PS at 48 weeks of treatment. Two children receiving teduglutide achieved enteral autonomy, after 12 weeks and 28 weeks of treatment, respectively. All adverse events (AEs) were in line with known impacts of SBS-IF and adverse reactions to teduglutide. Only one serious AE (abdominal pain) was considered related to teduglutide.

Conclusions: Short- and long-term treatment with teduglutide resulted in clinically meaningful reductions in PS requirements for infants and children with SBS-IF. Teduglutide was well tolerated, and efficacy improved with longer-term treatment.