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.