Safety/Tolerability of Glucagon-Like Peptide-2 Analog Apraglutide in Patients with Short Bowel Syndrome and Intestinal Failure: Phase 3 STARS trial

Background: Health utilities, a measure of health status required to conduct cost-utility analyses, are lacking in children with intestinal failure (IF).Current economic evaluations extrapolate utilities from adults.This study aims to estimate health utilities in children with IF on parenteral nutrition (PN) compared to children with IF that have achieved enteral autonomy (EA).Methods: Children with IF were recruited from two large intestinal rehabilitation centers in North America.Preference-based instruments were administered by interview to 50 children on PN and 57 children that achieved EA.Proxyassessments were conducted for children aged 5-7 years, while self-assessments were used for children aged 8-17 years.Utilities, ranging from 0 (death) to 1 (perfect health), were calculated using the Child Health Utility 9 dimensions (CHU9D) questionnaire.Differences in mean utilities between PN and EA groups were assessed with t-test.Results: For children aged 5-7 years, mean utilities on PN were 0.735 (SD 0.208), while those who achieved EA had mean utilities of 0.821 (SD 0.169), with no significant differences (p=0.269).For children aged 8-17 years, mean utilities on PN were 0.788 (SD 0.184), and mean utilities for EA were 0.838 (SD 0.142), with no significant differences (p=0.326).Conclusions: This multicenter study involving 107 children with IF provides the first estimation of pediatric health utilities.Estimated utilities for children with IF on PN are notably higher than published adult estimates (0.36 [SD 0.35]), while utilities of children that achieved EA align with adult estimates (0.82 [0.22]).These findings are crucial for advancing economic evaluations in pediatric IF.