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

## TL;DR

Teduglutide helps reduce the need for parenteral support in children with short bowel syndrome, showing real-world effectiveness over long-term use.

## Contribution

This study provides real-world evidence of teduglutide's long-term efficacy in reducing parenteral support dependency in pediatric short bowel syndrome patients.

## Key findings

- 67% of patients experienced a ≥20% reduction in parenteral support volume.
- 30% of patients achieved enteral autonomy within a mean of 71.57 weeks.
- Significant decreases in parenteral nutrition metrics were observed.

## Abstract

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.

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 %.

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.

Pediatric SBS patients experienced significant decreases in PS dependency, providing real-world evidence of teduglutide efficacy.