Holistic Mixed Approaches to Capture the Real Life of Children With Rare Eye Diseases

What this Trial Is About

Severe visual impairment and blindness in children caused by Rare Eye Diseases (RED) significantly affect their quality of life and psychological well-being. This research aims to understand the real-life impact of these conditions on children and teenagers, focusing on their personal experiences, family roles, and social participation. The study addresses the need for better tools that consider age and cultural differences to measure vision-related quality of life (VR-QoL) accurately and meet regulatory standards. The study uses a mixed methods research approach combining quantitative and qualitative techniques. Quantitative data are collected using validated questionnaires for children and teenagers (FVQ-CYP and VQoL-CYP) and caregiver questionnaires (PEM-CY) about participation and environment. Qualitative data involve semi-structured interviews, focus groups, and field observations to gain deeper insights into the children's daily lives and feelings about medical and social support. Participants will complete self-reported and caregiver questionnaires, take part in interviews and focus groups, and be observed in their living environments. The research will analyze all data to validate the questionnaires and develop a comprehensive VR-QoL tool. The main outcome measured is the real-life impact of severe visual impairment and blindness in children and teenagers over 12 months, providing valuable information for care, therapy development, and social integration. The study is led by University Hospital, Strasbourg.

Holistic Mixed Approaches to Capture the Real Life of Children With Rare Eye Diseases