Characteristics associated with response to teduglutide in pediatric short bowel syndrome-associated intestinal failure: Post hoc analysis of open-label long-term extension trials

Abstract

Background Teduglutide can reduce dependence on parenteral nutrition and/or intravenous fluids (PN/IV) in children with short bowel syndrome-associated intestinal failure (SBS-IF). Material and methods A post-hoc analysis was conducted using data from open-label long-term extension (LTE) trials (NCT02949362, NCT02954458) through 96 weeks. Patients were grouped by whether or not they attained: consistent response (≥ 20% PN/IV volume reduction from baseline for ≥ 90 consecutive days during both the parent and LTE studies); and sustained enteral autonomy (100% PN/IV volume reduction from baseline for ≥ 90 consecutive days at any point during 96 weeks of treatment). Univariate analysis identified factors associated with response to teduglutide. Results Overall, 23/69 patients had consistent response and 18/69 patients had sustained enteral autonomy. Significant differences were observed between those who did and did not attain consistent response in characteristics including age, presence of ileocecal valve, time since SBS diagnosis, and baseline PN/IV volume; and between patients with and without sustained enteral autonomy in age, length of remaining small intestine, and baseline PN/IV volume and duration. Older age at baseline and greater time since SBS diagnosis were associated with increased likelihood of consistent response (p < 0.05). Older age at baseline and greater length of remaining small intestine were associated with increased likelihood of sustained enteral autonomy (p < 0.05). Conclusions Of 69 children with SBS-IF, 33.3% had consistent response to teduglutide and 26.1% had sustained enteral autonomy. Age at baseline, time since diagnosis, and length of remaining small intestine were associated with response or enteral autonomy.