0766 Qualitative Development of the Pediatric Narcolepsy Patient-Reported Outcomes (PNPROS) Questionnaire

Abstract

Abstract Introduction There is a significant need to reflect patient needs and values in healthcare outcome measures. In pediatric narcolepsy, there are no patient-reported outcome measures to assess disease burden, determine treatment efficacy, and guide future drug development. The aim of this study was to develop items for pediatric narcolepsy patient-reported outcomes (PNPROS) measure. Methods A standardized approach for the development of patient-reported outcome measures was used to develop the PNPROS items, including domain and facet definitions; a systematic, comprehensive literature review; in-depth interviews with pediatric narcolepsy experts (n=3); and qualitative interviews with children and adolescents with narcolepsy ages 9-17 years (n=15) and parents of children/adolescents with narcolepsy (n=15). Once the preliminary item bank was created, iterative refinement was completed through another round of qualitative interviews with children/adolescents (n=11) and parents (n=11), as well as experts in pediatric sleep, narcolepsy, and measure development. Results The literature review and expert interviews contributed to the final conceptual framework, which included two domains. The first, Narcolepsy Symptoms, included 13 facets (behavior, brain fog, cataplexy, cognitive difficulties, difficulty waking, disrupted nighttime sleep, daytime sleepiness, hallucinations, mood, nightmares, parasomnias, sleep paralysis, and tired/fatigue). The second, Functioning Impairment, included 11 facets (bothered, brain fog, emotional/mood, home/family, lack of understanding, life satisfaction, safety, school/academic, social, weight gain, work/extracurriculars). The literature review identified 714 items from 27 measures; after redundancies or irrelevant items were removed, the 337 remaining items were assigned a domain and facet through team consensus. Qualitative interviews resulted in a further reduction of items due to lack of clarity or redundancy, resulting in the final 55 items to be included in field testing. Conclusion This rigorous qualitative approach to the development and evaluation of content validity for the PNPROS item pool resulted in 25 items that assess Narcolepsy Symptoms and 35 items that capture Functional Impairment. Once the psychometric properties are established, through field testing in a diverse, national sample of pediatric patients ages 9-17 years with narcolepsy, the measure will be useful for evaluating disease burden and treatment efficacy in both clinical and research settings. Support (if any) American Academy of Sleep Medicine Foundation