World journal of gastrointestinal pharmacology and therapeutics · 2025
Real-world effectiveness and safety of teduglutide in adult and pediatric patients with short bowel syndrome in Argentina.
СтатьяНазвание и аннотация: Перевод в очереди
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Solar Muñiz Héctor, Fernández Adriana, Busoni Verónica, Martínez María Inés, Rumbo Carolina, De Barrio Silvia, Saure Carola, Balacco Martín, Buncuga Martín Gonzalo, Dlugoszweski Corina, Manzur Alejandra, Rudi Lorena, Matoso María Dolores, Cosentino Sandra, Ussher Federico, Manzur Fernando, Demarchi Julia, Malaver Elisa, Brion Laura, Ungar Laura
Аннотация
BACKGROUND: Approval of teduglutide is an important addition to the limited treatment options for short bowel syndrome (SBS). However, real-world evidence on teduglutide therapy for SBS in Latin America is scarce.
AIM: To investigate the effectiveness and safety of teduglutide in clinical practice in Argentina with a 24-week follow-up.
METHODS: This non-interventional multicentre cohort study included consecutive patients (aged ≥ 1 years) with SBS who were dependent on parenteral support (PS) and received ≥ 1 dose of teduglutide according to currently approved indications.
RESULTS: The study population (n = 45) included 21 adult and 24 pediatric patients. The proportion of adult and pediatric patients who showed clinical response (defined as a ≥ 20% reduction in weekly PS volume) after 24 weeks of treatment was 90.4% [95% confidence interval (CI): 69.6%-98.8%] and 83.3% (95%CI: 62.6%-95.2%), respectively. Overall, 12 patients (26.6%; 95%CI: 14.6-41.9) were weaned from PS support at the 24-week assessment, 6 (28.5%; 95%CI: 11.5-52.1) in the adult cohort, and 6 (25%; 95%CI: 9.7-46.7) in the pediatric cohort. Only baseline PS requirement was inversely associated with weaning from PS (P = 0.025). The most frequently reported treatment-emergent adverse events (TEAEs) were mild to moderate abdominal pain and abdominal distension (16.6%; and 9.5%, respectively). None of the reported TEAEs led to treatment discontinuation.
CONCLUSION: This prospective real-world study demonstrated the effectiveness and safety of teduglutide in adult and pediatric patients with SBS in Argentina. The clinical response observed in both adults and pediatric patients was greater than that reported in phase 3 trials and was consistent with the results of other real-world studies.
AIM: To investigate the effectiveness and safety of teduglutide in clinical practice in Argentina with a 24-week follow-up.
METHODS: This non-interventional multicentre cohort study included consecutive patients (aged ≥ 1 years) with SBS who were dependent on parenteral support (PS) and received ≥ 1 dose of teduglutide according to currently approved indications.
RESULTS: The study population (n = 45) included 21 adult and 24 pediatric patients. The proportion of adult and pediatric patients who showed clinical response (defined as a ≥ 20% reduction in weekly PS volume) after 24 weeks of treatment was 90.4% [95% confidence interval (CI): 69.6%-98.8%] and 83.3% (95%CI: 62.6%-95.2%), respectively. Overall, 12 patients (26.6%; 95%CI: 14.6-41.9) were weaned from PS support at the 24-week assessment, 6 (28.5%; 95%CI: 11.5-52.1) in the adult cohort, and 6 (25%; 95%CI: 9.7-46.7) in the pediatric cohort. Only baseline PS requirement was inversely associated with weaning from PS (P = 0.025). The most frequently reported treatment-emergent adverse events (TEAEs) were mild to moderate abdominal pain and abdominal distension (16.6%; and 9.5%, respectively). None of the reported TEAEs led to treatment discontinuation.
CONCLUSION: This prospective real-world study demonstrated the effectiveness and safety of teduglutide in adult and pediatric patients with SBS in Argentina. The clinical response observed in both adults and pediatric patients was greater than that reported in phase 3 trials and was consistent with the results of other real-world studies.