Efficacy and Safety of Teduglutide in Infants and Children With Short Bowel Syndrome Dependent on Parenteral Support

Author:

Chiba Masahiro1,Masumoto Kouji2,Kaji Tatsuru3,Matsuura Toshiharu4,Morii Mayako5,Fagbemi Andrew6,Hill Susan7,Pakarinen Mikko P.8,Protheroe Susan9,Urs Arun10,Chen Szu-Ta11,Sakui Sho12,Udagawa Eri13,Wada Motoshi1415

Affiliation:

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

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

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

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

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

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

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

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

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

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

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

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

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

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

15. 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.

Publisher

Wiley

Subject

Gastroenterology,Pediatrics, Perinatology and Child Health

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