PMID- 37364133 OWN - NLM STAT- MEDLINE DCOM- 20230821 LR - 20230821 IS - 1536-4801 (Electronic) IS - 0277-2116 (Print) IS - 0277-2116 (Linking) VI - 77 IP - 3 DP - 2023 Sep 1 TI - Efficacy and Safety of Teduglutide in Infants and Children With Short Bowel Syndrome Dependent on Parenteral Support. PG - 339-346 LID - 10.1097/MPG.0000000000003867 [doi] AB - 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. CI - Copyright (c) 2023 The Author(s). Published by Wolters Kluwer on behalf of European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition. FAU - Chiba, Masahiro AU - Chiba M AD - From the Division of Pediatric Surgery, Department of Surgery, Showa University, Tokyo, Japan. FAU - Masumoto, Kouji AU - Masumoto K AD - the Department of Pediatric Surgery, Faculty of Medicine, University of Tsukuba, Tsukuba, Japan. FAU - Kaji, Tatsuru AU - Kaji T AD - the Department of Pediatric Surgery, Research Field in Medicine and Health Sciences, Medical and Dental Sciences Area, Research and Education Assembly, Kagoshima University, Kagoshima, Japan. FAU - Matsuura, Toshiharu AU - Matsuura T AD - the Department of Pediatric Surgery, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan. FAU - Morii, Mayako AU - Morii M AD - the Department of Pediatric Surgery, Akita University Graduate School of Medicine, Akita, Japan. FAU - Fagbemi, Andrew AU - Fagbemi A AD - the Department of Paediatric Gastroenterology, Royal Manchester Children's Hospital, Manchester, UK. FAU - Hill, Susan AU - Hill S AD - the Department of Paediatric Gastroenterology, Great Ormond Street Hospital for Children, NHS Foundation Trust and UCL Institute of Child Health, London, UK. FAU - Pakarinen, Mikko P AU - Pakarinen MP AD - the Pediatric Research Center, Children's Hospital, University of Helsinki, Helsinki University Hospital, Helsinki, Finland. FAU - Protheroe, Susan AU - Protheroe S AD - the Department of Gastroenterology and Nutrition, Birmingham Women and Children's Hospital, Birmingham, UK. FAU - Urs, Arun AU - Urs A AD - the Department of Paediatric Gastroenterology, Sheffield Children's Hospital, Sheffield, UK. FAU - Chen, Szu-Ta AU - Chen ST AD - Takeda Development Center Americas, Inc., Boston, MA, USA. FAU - Sakui, Sho AU - Sakui S AD - Statistical and Quantitative Sciences, Data Sciences Institute, Takeda Pharmaceutical Company Limited, Osaka, Japan. FAU - Udagawa, Eri AU - Udagawa E AD - Japan Medical Office, Takeda Pharmaceutical Company Limited, Tokyo, Japan. FAU - Wada, Motoshi AU - Wada M AD - the Department of Pediatric Surgery, Tohoku University Graduate School of Medicine, Sendai, Japan. AD - the Division of Pediatric Surgery, Department of Surgery, Tohoku University Hospital, Sendai, Japan. LA - eng SI - ClinicalTrials.gov/NCT03571516 SI - ClinicalTrials.gov/NCT03268811 SI - ClinicalTrials.gov/NCT02980666 PT - Journal Article PT - Randomized Controlled Trial PT - Research Support, Non-U.S. Gov't DEP - 20230626 PL - United States TA - J Pediatr Gastroenterol Nutr JT - Journal of pediatric gastroenterology and nutrition JID - 8211545 RN - 7M19191IKG (teduglutide) RN - 0 (Peptides) RN - 0 (Gastrointestinal Agents) SB - IM MH - Humans MH - Infant MH - Child MH - *Short Bowel Syndrome/drug therapy MH - Parenteral Nutrition/methods MH - Intestine, Small MH - Peptides/adverse effects MH - Gastrointestinal Agents/adverse effects PMC - PMC10412081 COIS- S.C. is an employee of Takeda Pharmaceutical Company Limited, Cambridge, MA, USA and is a stockholder of Takeda Pharmaceutical Company Limited. S.S. is an employee of Takeda Pharmaceutical Company Limited, Osaka, Japan and is a stockholder of Takeda Pharmaceutical Company Limited. E.U. was an employee of Takeda Pharmaceutical Company Limited, Tokyo, Japan. M.W. received research funding from Shire, a Takeda company. The remaining authors report no conflicts of interest. EDAT- 2023/06/26 19:07 MHDA- 2023/08/21 06:42 PMCR- 2023/08/09 CRDT- 2023/06/26 15:13 PHST- 2023/08/21 06:42 [medline] PHST- 2023/06/26 19:07 [pubmed] PHST- 2023/06/26 15:13 [entrez] PHST- 2023/08/09 00:00 [pmc-release] AID - 00005176-990000000-00422 [pii] AID - 10.1097/MPG.0000000000003867 [doi] PST - ppublish SO - J Pediatr Gastroenterol Nutr. 2023 Sep 1;77(3):339-346. doi: 10.1097/MPG.0000000000003867. Epub 2023 Jun 26.