BACKGROUND: With significant reductions in morbidity and mortality for children with pediatric intestinal failure (PIF), assessing long-term patient quality of life (QoL) has become a priority. However, the lack of a PIF-specific QoL assessment tool has impeded investigators’ ability to measure QoL holistically and meaningfully across a range of domains relevant to this patient population. The purpose of this study was to develop a disease-specific questionnaire to assess QoL in children with PIF and apply this tool in a longitudinal study at a large intestinal rehabilitation program (IRP).
METHODS: Using a community-driven research design, a team of patient, parent, and clinician researchers developed a PIF-specific QoL tool to measure how children feel about- and whether they are bothered by- 42 Likert-scale items. Scores range from 0-100 with the middle range being neutral and scores below or above indicating negative or positive QoL orientations respectively. The tool was applied in a longitudinal study; PIF patients ages 8-18 completed the survey on a tablet at outpatient IRP clinic visits. Descriptive analyses were conducted; results are presented as means and percentages.
RESULTS: Initial instrument assessment suggests excellent construct and face validity and adequate internal consistency and reliability. The instrument was well-tolerated in the clinic context, with patients across the eligible age range able to complete the assessment independently in ~10 minutes. Fifty-six child self-report questionnaires were completed at Time 1 with a mean patient age of 13.5 years. Of the respondents, 28 (51 percent) were receiving parenteral support and 9 (17 percent) had an ostomy at the time of assessment. The mean total instrument score was 67.9, suggesting a good average self-reported QoL for patients in the sample. Domain and sub-domain scores varied. The lowest scores were reported in the “medical devices and therapies” sub-domain (mean=52.1), with 45 percent of children reporting a score below 45, indicating they were slightly to very unhappy about their respective medical devices or therapies. The remaining 55 percent reported feeling neutral to positive about the items within this domain. The highest domain scores were achieved in the “social” (mean=77.5) and “school” (mean=72.8) domains and the “self-management” (mean=69.5) sub-domain, with 91, 83, and 79 percent of children respectively reporting scores of 55 or above, suggesting they were slightly to very happy about items within these domains. In preliminary univariate analyses, no statistically significant differences in mean domain scores were observed between children on and off parenteral support.
CONCLUSION: Results indicate QoL within this patient cohort is multifaceted and generally positive. Across all survey domains and subdomains, most children did not report feeling unhappy or bothered; they were most happy about their social and school lives and their ability to self-manage their condition. Future work will compare child self-reported QoL with parent proxy reports and will aim to identify clinical or socio-demographic factors associated with PIF-specific QoL. Applying this tool at other centers would allow for QoL comparisons across institutions.
