ERNICA Research Collaboration Webinar on Quality of Life

Health-related quality of life (HRQoL) is a narrower concept than general QoL, relating specifically to the individual's perception of the impact of disease and treatment on physical, social, and psychological functioning and well-being.

Generic HRQoL assessments allow comparison between healthy people and patients; condition-specific assessments are more sensitive to treatment response but do not permit external comparison.

Most development of QoL and HRQoL concepts has occurred in the last few decades, with WHO defining three dimensions of health in 1948 and HRQoL entering clinical research in the late 1980s–early 1990s.

PROM development follows six steps: literature review, qualitative elicitation (interviews/focus groups), item drafting, cognitive debriefing/pilot testing, item revision, and psychometric evaluation (validity, reliability, responsiveness).

Cross-cultural PROM validation requires linguistic/semantic equivalence (correct translation), conceptual equivalence (similar domain importance across cultures), and measurement equivalence (same construct measured, comparable scores).

Generic HRQoL instruments (PedsQL, CHQ, SF-36) typically cover physical, emotional/mental, and social functioning domains.

The DCGM-25 (DISABKIDS Chronic Generic Measure) uniquely focuses on the emotional process of satisfaction, a more subjective process, in addition to functional appraisal.

Proxy ratings (parent reports) and self-ratings correlate but not highly, indicating they represent two distinct perspectives on the patient's life satisfaction.

The Haquel (Hirschsprung disease and anorectal malformation quality of life questionnaire) was developed in the Netherlands over 20 years ago, has acceptable-to-good internal consistency (Cronbach's alpha), discriminates between disease-severity subgroups, and shows substantial but not excessive correlation with the generic SF-36.

The EA-QoL (esophageal atresia quality of life) questionnaires followed a rigorous development process: qualitative research, cross-cultural item generation, translation into 15 countries, and cognitive debriefing to ensure parallel cultural content.

The SCIA (adult esophageal atresia questionnaire) was developed because the SF-36 and existing gastrointestinal/respiratory symptom indexes did not capture the full range of health problems in adults with EA.

Benefits of PROM use include bringing daily life into the hospital, improving care by prompting patients to contemplate important issues at home, and enabling comparison of clinical groups or intervention outcomes in research.

Costs of PROM use include patient burden (adolescents often dislike questionnaires), some patients' belief that questionnaires cannot fully capture their lived experience, the time required for interpretation and motivation, and lack of visible benefit when clinicians do not discuss results in consultation.

The DCGM-25 uses five smiley-face response options (two sad, one neutral, two happy) instead of verbal labels, making it easier for children with language-processing difficulties, non-native speakers, or intellectual disabilities to complete.

In rare pediatric-surgery conditions, cultural validation is complicated because once you collect a population in one country, clinical characteristics may differ from another country's cohort, making it difficult to separate cultural from clinical influences on QoL perception.