Your browser doesn't support javascript.
loading
Efficacy and Safety of Teduglutide in Infants and Children With Short Bowel Syndrome Dependent on Parenteral Support.
Chiba, Masahiro; Masumoto, Kouji; Kaji, Tatsuru; Matsuura, Toshiharu; Morii, Mayako; Fagbemi, Andrew; Hill, Susan; Pakarinen, Mikko P; Protheroe, Susan; Urs, Arun; Chen, Szu-Ta; Sakui, Sho; Udagawa, Eri; Wada, Motoshi.
Afiliação
  • Chiba M; From the Division of Pediatric Surgery, Department of Surgery, Showa University, Tokyo, Japan.
  • Masumoto K; the Department of Pediatric Surgery, Faculty of Medicine, University of Tsukuba, Tsukuba, Japan.
  • Kaji T; 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.
  • Matsuura T; the Department of Pediatric Surgery, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan.
  • Morii M; the Department of Pediatric Surgery, Akita University Graduate School of Medicine, Akita, Japan.
  • Fagbemi A; the Department of Paediatric Gastroenterology, Royal Manchester Children's Hospital, Manchester, UK.
  • Hill S; the Department of Paediatric Gastroenterology, Great Ormond Street Hospital for Children, NHS Foundation Trust and UCL Institute of Child Health, London, UK.
  • Pakarinen MP; the Pediatric Research Center, Children's Hospital, University of Helsinki, Helsinki University Hospital, Helsinki, Finland.
  • Protheroe S; the Department of Gastroenterology and Nutrition, Birmingham Women and Children's Hospital, Birmingham, UK.
  • Urs A; the Department of Paediatric Gastroenterology, Sheffield Children's Hospital, Sheffield, UK.
  • Chen ST; Takeda Development Center Americas, Inc., Boston, MA, USA.
  • Sakui S; Statistical and Quantitative Sciences, Data Sciences Institute, Takeda Pharmaceutical Company Limited, Osaka, Japan.
  • Udagawa E; Japan Medical Office, Takeda Pharmaceutical Company Limited, Tokyo, Japan.
  • Wada M; the Department of Pediatric Surgery, Tohoku University Graduate School of Medicine, Sendai, Japan.
J Pediatr Gastroenterol Nutr ; 77(3): 339-346, 2023 09 01.
Article em En | MEDLINE | ID: mdl-37364133
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.
Assuntos

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Síndrome do Intestino Curto Tipo de estudo: Clinical_trials Limite: Child / Humans / Infant Idioma: En Ano de publicação: 2023 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Síndrome do Intestino Curto Tipo de estudo: Clinical_trials Limite: Child / Humans / Infant Idioma: En Ano de publicação: 2023 Tipo de documento: Article