Measuring What Matters: A Cross-Sectional Assessment of Disease-Specific Self- and Proxy-Reported Quality of Life in Pediatric Intestinal Failure

With greatly improved clinical outcomes in pediatric intestinal failure (PIF), the assessment of non-clinical, patient-reported outcomes (PROs) has become increasingly prioritized. One such outcome is patient quality of life (QoL). A growing body of work has investigated PIF patient QoL using generic assessment tools, such as the PedsQL, which measures patients’ health-related functioning. While there are nuances, this line of research has generally provided evidence of diminished health-related QoL. However, the voices of patients and caregivers in this research have largely been missing, and persistent knowledge gaps about QoL in PIF remain. Our multidisciplinary group of scholars previously developed and validated a PIF-specific QoL assessment tool using a community-driven research design. In the present study, the PIF-QoL was administered at the University of Nebraska Medical Center, a leading center providing multidisciplinary PIF care, to provide baseline disease-specific QoL data for this patient population and investigate the connection between select clinically relevant factors and patient QoL. Descriptive analyses were conducted to provide baseline self- and proxy-reported disease-specific QoL data. Univariate analyses compared child- and caregiver-reported scores, as well as QoL scores reported by male and female caregivers, to examine differences by respondent type. Finally, a causal inference approach was used to assess the causal association of five clinically relevant factors with patient QoL. Seventy-five patients and 200 caregivers completed the self- and proxy-report versions of the PIF-QoL, respectively. Mean QoL scores in both samples were above the mid-range, suggesting that, on average, children were happy with their QoL across all assessed domains. Compared to their caregivers, children self-reported higher mean scores in domains related to their IF management and in the instrument summary score. Female caregivers reported significantly lower scores than male caregivers in select domains and the instrument summary score. Causal analyses suggest that longer time spent receiving UNMC IRP management was associated with higher QoL, whereas enteral nutrition was associated with lower QoL. Together, these findings present a powerful counterstory to the dominant deficit-oriented, biomedical narrative about QoL in PIF, suggesting that challenges and limitations coexist with resilience and positive well-being.