Real-world efficacy of long-term teduglutide use in pediatric patients with short bowel syndrome.

Background: In patients with short bowel syndrome (SBS), teduglutide reduces dependency on parenteral support (PS) by promoting intestinal growth and absorption. We aim to describe long-term outcomes using teduglutide in a large pediatric intestinal rehabilitation and transplant center. Material and Methods: We performed a single-center, retrospective analysis of teduglutide use in patients with SBS ages 1-23 years. Sex, age, intestinal length, BMI, PS regimens, and isolated small bowel (ISB) transplant status are described. Subgroup analysis comparing younger and older patient cohorts was performed. Primary end point was a reduction in PS volume of ≥ 20 %. Results: 27 patients (10 female; mean age 8.9 years) received subcutaneous teduglutide (0.05 mg/kg/d) once daily for mean 120.8 weeks. Mean bowel length was 56.9 (SD=52.1) cm. 5 were listed for ISB transplant, 2 were "inactive," and 20 were not listed. 2 patients were post-enterectomy. A decrease in PS volume of ≥ 20 % was experienced by 67 % of patients, with 30 % reaching enteral autonomy at mean 71.57 (SD=56.37) weeks (Fig. 1). Significant decreases in PS volume, hours and days, nonprotein calories, parenteral nutrition dependency index (PNDI), and lipid dose were observed (Table 2). Of patients listed at baseline, 2 remained listed, 2 were removed, and 1 was reclassified as "inactive." 1 was newly listed and 2 "inactive" patients were removed. 19 remained unlisted.The most frequent adverse event was pyrexia. 10 patients permanently discontinued treatment at mean 62.59 (SD=55.55) weeks, 4 due to related AEs. Conclusion: Pediatric SBS patients experienced significant decreases in PS dependency, providing real-world evidence of teduglutide efficacy.