Outcomes and adverse effects of teduglutide treatment with periodic withdrawal in pediatric short bowel patients

OBJECTIVES: We evaluated the efficacy and safety of teduglutide in a real-world cohort in which national reimbursement policies required treatment interruptions. The primary outcomes were reduction in parenteral support (PS) and treatment-related adverse effects, and the secondary outcome was the impact of the mandated withdrawal periods. METHODS: In this retrospective registry-based nationwide study, we identified all pediatric short bowel-associated intestinal failure (IF) patients treated with teduglutide and reviewed clinical records to evaluate PS reduction and adverse effects during treatment and withdrawal. RESULTS: Twenty pediatric IF patients had teduglutide initiated (age range: 1.4-17 years) between 2016 and 2022. Patients had a median 16% small bowel remaining, and 16 (79%) had the ileocecal valve removed. Children who continued teduglutide over 6 months (n = 19) had a median of 31 (range: 13-56) months of follow-up on medication. Their median reductions in daily PS energy and fluid volume requirement were 42% and 28 mL/kg/day, respectively. Weekly infusion-free nights increased from 0.5 to 4. Seventeen (89%) patients reached >20% energy reduction. Six patients (32%) successfully weaned off PS. Four patients (20%) experienced intestinal bleeding while on teduglutide. Stomal prolapses occurred in all patients with end-jejunostomies. Periodic withdrawal (n = 37) led to adverse effects on 84% of occasions (n = 32), including two unplanned hospitalizations and 14 (38%) outpatient visits, dehydration in 19 (51%), and diarrhea in 28 (76%) occasions. CONCLUSIONS: Teduglutide resulted in clinically significant PS reduction and increased infusion-free nights. The periodic withdrawal of teduglutide was harmful and should not be routinely employed. TRIAL REGISTRATION: ClinicalTrials.gov identifier: HUS/265/2023.