Journal of pediatric gastroenterology and nutrition · 2026
Quality of life for pediatric patients with short bowel syndrome-associated intestinal failure treated with teduglutide.
СтатьяРандомизированное исследованиеКлиническое исследование III фазыНазвание и аннотация: Перевод в очереди
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Wendel Danielle, Wales Paul W, Kocoshis Samuel, Venick Robert, Carter Beth, Cohran Valeria, Matthews Cheney, Raphael Bram P, Robinson Ian, Hill Susan
Аннотация
OBJECTIVES: The complex care needs of pediatric patients with short bowel syndrome-associated intestinal failure (SBS-IF) can negatively impact the health-related quality of life (HRQoL) of patients and their caregivers. We assessed the impact of teduglutide on HRQoL of pediatric patients with SBS-IF.
METHODS: Two long-term extension (LTE) studies (NCT02949362, NCT02954458) assessed HRQoL over 96 weeks using the Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales and the PedsQL Family Impact Module. Patients included in this analysis received teduglutide in one of three randomized Phase 3 parent studies and the respective LTE study. We assessed changes in HRQoL over time, as well as the impact of attaining enteral autonomy on HRQoL and predictors of change in HRQoL.
RESULTS: In pediatric patients with SBS-IF receiving teduglutide (n = 69), the total mean (standard deviation) patient-reported PedsQL score was 75.0 (16.2). Higher mean number of stools per day at LTE study baseline (p = 0.03) predicted increased patient-reported HRQoL. Longer teduglutide exposure (p = 0.047), increasing length of remnant small intestine (p = 0.034), and older age (p = 0.026) predicted higher caregiver-proxy-reported HRQoL. Mean PedsQL scores remained stable over 96 weeks regardless of patients achieving enteral autonomy.
CONCLUSIONS: HRQoL of pediatric patients with SBS-IF treated with teduglutide was generally stable over 96 weeks. Predictors of improved HRQoL included higher baseline stool number, longer teduglutide treatment duration, greater length of small intestine, and older age. To better understand these observations, future studies should assess HRQoL before initiation of treatment.
CLINICAL TRIAL REGISTRATION: Clinical trials NCT02949362 and NCT02954458.
METHODS: Two long-term extension (LTE) studies (NCT02949362, NCT02954458) assessed HRQoL over 96 weeks using the Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales and the PedsQL Family Impact Module. Patients included in this analysis received teduglutide in one of three randomized Phase 3 parent studies and the respective LTE study. We assessed changes in HRQoL over time, as well as the impact of attaining enteral autonomy on HRQoL and predictors of change in HRQoL.
RESULTS: In pediatric patients with SBS-IF receiving teduglutide (n = 69), the total mean (standard deviation) patient-reported PedsQL score was 75.0 (16.2). Higher mean number of stools per day at LTE study baseline (p = 0.03) predicted increased patient-reported HRQoL. Longer teduglutide exposure (p = 0.047), increasing length of remnant small intestine (p = 0.034), and older age (p = 0.026) predicted higher caregiver-proxy-reported HRQoL. Mean PedsQL scores remained stable over 96 weeks regardless of patients achieving enteral autonomy.
CONCLUSIONS: HRQoL of pediatric patients with SBS-IF treated with teduglutide was generally stable over 96 weeks. Predictors of improved HRQoL included higher baseline stool number, longer teduglutide treatment duration, greater length of small intestine, and older age. To better understand these observations, future studies should assess HRQoL before initiation of treatment.
CLINICAL TRIAL REGISTRATION: Clinical trials NCT02949362 and NCT02954458.