Health-Related Quality of Life in Pediatric Intestinal Failure from Infancy to Adolescence: An International, Multi-Center Evaluation

Abstract

Objective: To evaluate health-related quality of life (HRQOL) and associated factors in pediatric intestinal failure (PIF) using a prospective, multi-institutional structure.

Study design: HRQOL in 336 children with PIF was assessed using the PedsQL Generic Core and Gastrointestinal (GI) Symptoms Scales at 11 North American intestinal rehabilitation programs. Scores were compared between patients, caregivers' proxy report of patient HRQOL, and reference healthy and chronic GI disease samples. Multivariable linear regression assessed factors associated with HRQOL.

Results: Adolescents reported significantly higher PedsQL (absolute difference [95% CI] 10 [4.9, 15.2]) and GI Symptoms Scales (5.9 [1, 10.7]) scores than caregivers. Patients and caregivers reported lower HRQOL but higher GI Symptoms Scales scores compared with reference samples. On multivariable analysis, neurologic comorbidity had the strongest negative impact on HRQOL for patients (adjusted coefficient -12.3 [95% CI -21.5, -3.1]) and caregivers (-11.9 [-17.7, -6.2]), and caregivers of 8-12-year-olds independently reported the lowest HRQOL scores (-13.5 [-23, -3.9]). Clinical factors with negative impact on HRQOL included ostomy presence (-11.4) and hospitalizations (-7.9) for caregivers and emergency department visits (-10.1) for patients.

Conclusions: This large, multicenter study provides benchmark data for HRQOL in PIF. Adolescents reported a higher HRQOL than caregivers. HRQOL in PIF was lower than reference samples. Multiple independent factors with negative impact on HRQOL were identified. Future studies will focus on longitudinal HRQOL trends, impact on families, and interventions targeting risk factors to optimize long-term HRQOL.

Clinical trial: Although this is currently an observation only study, it was registered through the National Library of Medicine at ClinicalTrials.gov as "Prospective Multisite Study of Quality of Life in Pediatric Intestinal Failure", study ID NCT04629014.