Measuring disease-specific quality of life in pediatric intestinal failure: Development and validation of the PIF-QoL

Research suggests pediatric intestinal failure (PIF) patients experience impaired quality of life (QoL), but no disease-specific instrument has been available to assess this patient population. The aim of this study was to develop and validate a PIF-specific QoL assessment tool.

Our team of patient-, caregiver-, and clinician researchers developed a questionnaire to assess self- and proxy-reported QoL in PIF using a community-driven study design that included a literature review and exploratory study, review by content experts, focus groups and cognitive interviews with children, and the tool's implementation at a large U.S.-based PIF center. An initial psychometric evaluation was conducted to assess validity and reliability.

A total of 181 caregivers of children (ages 4-18) with a history of PIF and 70 patients (ages 8-18) with a history of PIF completed the caregiver and child versions of the PIF-QoL, respectively, and were included in the internal consistency assessment. Test-retest reliability was assessed with a sample of 30 caregivers and 21 children who completed the questionnaires at two timepoints. Across both PIF-QoL versions, results suggest acceptable to good internal consistency and test-retest reliability for individual domains. One exception was the "Food and Eating" domain, which may warrant future refinement. Instrument internal consistency and test-retest reliability were excellent (Caregiver: α≥ 0.89, ICC= 0.90; Child: α≥ 0.83, ICC= 0.92).

Initial empirical testing and psychometric evaluation of the PIF-QoL support its use as a practical, valid, and reliable tool for assessing self- and proxy-reported QoL of patients with PIF.