Safety Findings in Pediatric Patients During Long-Term Treatment With Teduglutide for Short-Bowel Syndrome-Associated Intestinal Failure: Pooled Analysis of 4 Clinical Studies.

Abstract

BackgroundThis analysis assessed combined safety data from 4 clinical studies of teduglutide in pediatric patients with short‐bowel syndrome–associated intestinal failure (SBS–IF).MethodsSafety data from teduglutide‐treated patients in 4 clinical trials were pooled. The completed 12‐week and 24‐week phase 3 core studies (NCT01952080/EudraCT 2013‐004588‐30 and NCT02682381/EudraCT 2015‐002252‐27) enrolled children aged 1–17 years with SBS–IF. Patients could elect to enroll in ongoing open‐label extensions (NCT02949362/EudraCT 2016‐000863‐17 and NCT02954458/EudraCT 2016‐000849‐30). Interim data from ongoing studies were included.ResultsSafety data are reported for 89 pediatric patients treated with teduglutide for a median (range) of 51.7 (5.0–94.7) weeks. Adverse events (AEs) were reported in all patients; the most common were vomiting (51.7%), pyrexia (43.8%), upper respiratory tract infection (41.6%), and cough (33.7%). Thirty‐five patients (39.3%) had AEs considered related to teduglutide treatment; abdominal pain and vomiting were most frequent (5.6% each). Three serious AEs in 3 patients (3.4%) were considered related to teduglutide treatment: ileus, d‐lactic acidosis, and gastrointestinal obstruction due to hard stools. All 3 events resolved. One cecal polyp was detected, which was not biopsied or found on repeat colonoscopy. No cases of neoplasia occurred.ConclusionBased on integrated data from 4 clinical studies, including long‐term follow‐up for ≤161 weeks, teduglutide had a safety profile consistent with the individual core pediatric studies and as expected for pediatric patients with SBS–IF who never received teduglutide. The most frequent AEs reflected treatment with teduglutide, complications of the underlying disease, and typical childhood illnesses.