Safety Findings in Pediatric Patients During Long-Term Treatment With Teduglutide for Short-Bowel Syndrome-Associated Intestinal Failure: Pooled Analysis of 4 Clinical Studies.

Autor: Hill S; Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Carter BA; Children's Hospital Los Angeles and Keck School of Medicine, University of Southern California, Los Angeles, California, USA., Cohran V; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA., Horslen S; Seattle Children's Hospital and University of Washington School of Medicine, Seattle, Washington, USA., Kaufman SS; MedStar Georgetown University Hospital, Washington, DC, USA., Kocoshis SA; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA., Mercer DF; University of Nebraska Medical Center, Omaha, Nebraska, USA., Merritt RJ; Children's Hospital Los Angeles and Keck School of Medicine, University of Southern California, Los Angeles, California, USA., Pakarinen MP; Children's Hospital, University of Helsinki, Helsinki, Finland., Protheroe S; Birmingham Children's Hospital NHS Foundation Trust, Birmingham, UK., Thompson JF; Children's Hospital at Montefiore, Bronx, New York, USA., Vanderpool CPB; Riley Hospital for Children, Indianapolis, Indiana, USA., Venick RS; Mattel Children's Hospital UCLA California, Los Angeles, California, USA., Wales PW; The Hospital for Sick Children, Toronto, Ontario, Canada., Smith SE; Shire Human Genetic Therapies, Inc, a Takeda company, Cambridge, Massachusetts, USA., Yoon M; Shire Human Genetic Therapies, Inc, a Takeda company, Cambridge, Massachusetts, USA., Grimm AA; Shire Human Genetic Therapies, Inc, a Takeda company, Cambridge, Massachusetts, USA.
Jazyk: angličtina
Zdroj: JPEN. Journal of parenteral and enteral nutrition [JPEN J Parenter Enteral Nutr] 2021 Sep; Vol. 45 (7), pp. 1456-1465. Date of Electronic Publication: 2021 Mar 02.
DOI: 10.1002/jpen.2061
Abstrakt: Background: This analysis assessed combined safety data from 4 clinical studies of teduglutide in pediatric patients with short-bowel syndrome-associated intestinal failure (SBS-IF).
Methods: Safety data from teduglutide-treated patients in 4 clinical trials were pooled. The completed 12-week and 24-week phase 3 core studies (NCT01952080/EudraCT 2013-004588-30 and NCT02682381/EudraCT 2015-002252-27) enrolled children aged 1-17 years with SBS-IF. Patients could elect to enroll in ongoing open-label extensions (NCT02949362/EudraCT 2016-000863-17 and NCT02954458/EudraCT 2016-000849-30). Interim data from ongoing studies were included.
Results: Safety data are reported for 89 pediatric patients treated with teduglutide for a median (range) of 51.7 (5.0-94.7) weeks. Adverse events (AEs) were reported in all patients; the most common were vomiting (51.7%), pyrexia (43.8%), upper respiratory tract infection (41.6%), and cough (33.7%). Thirty-five patients (39.3%) had AEs considered related to teduglutide treatment; abdominal pain and vomiting were most frequent (5.6% each). Three serious AEs in 3 patients (3.4%) were considered related to teduglutide treatment: ileus, d-lactic acidosis, and gastrointestinal obstruction due to hard stools. All 3 events resolved. One cecal polyp was detected, which was not biopsied or found on repeat colonoscopy. No cases of neoplasia occurred.
Conclusion: Based on integrated data from 4 clinical studies, including long-term follow-up for ≤161 weeks, teduglutide had a safety profile consistent with the individual core pediatric studies and as expected for pediatric patients with SBS-IF who never received teduglutide. The most frequent AEs reflected treatment with teduglutide, complications of the underlying disease, and typical childhood illnesses.
(© 2020 American Society for Parenteral and Enteral Nutrition.)
Databáze: MEDLINE
Externí odkaz: