Real-world effectiveness and safety of teduglutide in adult and pediatric patients with short bowel syndrome in Argentina

Abstract

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.

Keywords: Short bowel syndrome; Intestinal failure; Parenteral nutrition; Parenteral support; Weaning; Teduglutide; Real-world

Core Tip: This multicenter real-world evidence study demonstrates the effectiveness and safety of teduglutide in adult and pediatric patients with short bowel syndrome-IF in the Latin American population. The results showed that teduglutide induced significant reductions in parenteral support (PS) in both adult and pediatric populations, consistent with pivotal studies, with a low incidence of mild to moderate adverse events. The findings highlight the importance of early identification of eligible patients for teduglutide (patients with reduced baseline PS volume dependence and/or favorable bowel anatomy), which may increase the likelihood of achieving PS independence. This approach could potentially reduce the morbidity associated with intestinal failure, ultimately improving long-term patient outcomes in Latin America.