Development of Patient Reported Outcomes System for Children and Young Adults with pNF using Mixed Method Approach

Although health-related quality of life (HRQOL) is compromised for children with pNF across all aspects of well-being, based on a review of the literature and clinical observations, our study team identified stigma and self-concept as the main psychological adjustment issues regarding children with pNF and pain as the main symptom that impacts their HRQOL. Though the final instrument domains to be developed will be further determined by qualitative interview results, we anticipate that pain and stigma will be among the top concerns reported by interviewees. We plan to modify related existing instruments from two national efforts: Patient Reported Outcomes Measurement Information System (PROMIS) and Quality of Life in Neurological Disorders (Neuro-QOL), given their comprehensive coverage of domains and the state-of-art development and validation approaches. Additionally, both of them were developed flowing the FDA guideline, specifically individual’s voices were well-integrated in the item development process