Development of a Pediatric Narcolepsy Patient-Reported Outcomes Scale
Background: Pediatric narcolepsy is a chronic neurological disorder that impacts the health and overall well-being of children and adolescents with the disease. Meaningful and regular assessment of symptom frequency and severity is important for the long-term management of narcolepsy and for optimizing quality of life. However, there is currently no patient-reported outcomes (PROs) measure developed from both patient and expert input that is designed specifically to assess the impact of pediatric narcolepsy on daily life and overall disease burden. Methods: We conducted a qualitative mixed-methods study to develop a novel patient-reported outcomes measure for pediatric narcolepsy patients. We created and refined a conceptual framework through literature review, semi-structured interviews with narcolepsy experts, and focus groups of children and adolescents with narcolepsy and their parents. Guided by the domains and facets identified in our conceptual framework and further literature r