Long-term Durability Between Parent and Child Patient-Reported Outcomes in Eosinophilic Esophagitis

To assess the agreement and stability of patient-reported outcomes (PRO) between children with eosinophilic esophagitis (EoE) and their parents using longitudinal datasets.Children aged between 3 and 18 years with EoE and their parents.Between 2015 and 2021, 2 questionnaires were administered on entry and at follow-up visits. In children aged 8 to 17 years, the Pediatric Eosinophilic Esophagitis Symptom Score version 2 (PEESSv2.0) considers symptom frequency and severity. The parent version (for children aged 2–17 years) domains are subjective (eg, frequency of difficulty swallowing) and observable (eg, severity of difficulty swallowing) measures. PEESSv2.0 has a score of 0 to 100 with higher scores being worse. The Pediatric Quality of Life Eosinophilic Esophagitis Module (PedsQL-EoE) domains are symptoms, problems with treatment, worry, communication, and food/eating. The PedsQL-EoE assesses young children (5–7 years), children (8–12 years), and adolescents (13–18 years). The parent version is for those whose children are aged 2 to 4, 5 to 7, 8 to 12, and 13 to 18 years. It is scored from 0 to 100 with higher scores indicating better quality of life (QoL).Parent-child pairs (n = 292) completed 723 questionnaires over 5 years with 64% completing at least 2 surveys. The median age of onset was 5 years, and the median duration of disease before entry was 3.7 years. No changes were found over time in parent or child self-reported PEESSv2.0 (P ≥ .43). Black children self-reported more symptoms (17.9 points higher, P = .002) than white children, and girls self-reported more symptoms than boys (5.9 points higher, P = .019). Because Food and Drug Administration guidance focuses on the observable (severity) domain of PEESSv2.0, the authors chose to assess parental reports of the subjective (frequency) domain. There was a strong correlation between it and the total PEESSv2.0 score for both parent and child reports (r = 0.97). Neither parent nor child frequency scores changed over time. Higher scores were reported by girls than boys (P = .019) and by Black children than white children (P = .005). Parents’ PedsQL-EoE score did not change over time, but children’s QoL improved over time, as reflected by their self-report total score (P = .026). Girls and their parents reported lower PedsQL-EoE scores than boys and their parents (P = .023). Self-report PedsQL-EoE scores were lowest for young children and higher for adolescents (P < .001). Group-based analysis of PRO trajectory over time used total PEESSv2.0 score. Three groups (scores >50, around 30, and <20) were identified, matching clinically suggested score cutoffs for severe, moderate, and mild symptoms. Parent and child reports were stable over the study period, but parents reported fewer severe scores and more scores in the mild range than their children (49% vs 63% and 16% vs 4%, respectively). Trajectory analysis for PedsQL-EoE also identified 3 groups for parent and child self-reports with mean scores of approximately 80, 60, and 40. Children with low and moderate scores improved over time (P < .001 for both). In contrast, parent PedsQL-EoE scores were unchanged in all 3 groups and remained comparable to their children’s scores at entry. Finally, the authors assessed parent/child intersubject correlations with strong correlation for PEESSv2.0 (r = 0.83), PEESSv2.0 frequency score (r = 0.83), and PedsQL-EoE (r = 0.79), although the parental PEESSv2.0 frequency and PedsQL-EoE scores were higher than their children’s (P = .04 and P = .029, respectively).EoE PROs of children and parents are aligned over time. Parental reports are a stable proxy for objective EoE symptoms in their children.Because young children cannot describe the severity and quality of their symptoms, we rely on parental surrogate reports. This study demonstrates that for EoE, parents are good reporters of their children’s symptoms and QoL. A possible exception reported herein is that despite parents agreeing with the children who self-reported low and moderate PedsQL-EoE scores at entry, they did not observe the same improvement in QoL at subsequent visits (Reviewer’s aside: As pediatricians, do we expect tweens and teens to agree on everything with their parents?). We cannot extrapolate these data to assume that parents provide accurate proxy histories for other chronic or acute conditions, but they are encouraging. Similar studies of objective and subjective symptoms (including QoL) in other immunologic and nonimmunologic diseases will likely be insightful.