Objectives: Neurofibromatosis (NF) is a genetic disease with multiple clinical manifestations that can significantly impact quality of life (QOL). Clinical trials should include patient-reported outcomes (PROs) as endpoints to assess treatment effects on various aspects of QOL, but there is no consensus on the selection and use of such measures in NF. This article describes the PRO Working Group of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) Collaboration, its main goals, methods for identifying appropriate PRO measures for NF clinical trials, and recommendations for assessing pain intensity.Methods: The REiNS PRO group selected core endpoint domains important to assess in NF. The members developed criteria to rate PRO measures, including patient characteristics, psychometric properties, and feasibility, and utilized a systematic process to evaluate PROs for NF clinical trials. Within the subdomain of pain intensity, the group reviewed the Numerical Rating Scale-11 (NRS-11), the Visual Analogue Scale, and the Faces Pain Scale-Revised using this process.Results: Based on the review criteria, each of these pain intensity scales is brief, reliable, valid, and widely used. However, the NRS-11 was given the highest rating for use in NF clinical trials due to recommendations from pain experts and other consensus groups, its extensive use in research, strong psychometric data including sensitivity to change, and excellent feasibility in ages $8 years.
Conclusions:The systematic review criteria and process are effective for identifying appropriate PRO measures and provide information utilized by the REiNS Collaboration to achieve consensus regarding PROs in NF clinical trials. Neurology ® 2013;81 (Suppl 1):S6-S14
Background: Down syndrome has a unique medical and psychological profile. To date, few studies have asked individuals with Down syndrome about their views of health.Methods: Eight focus groups of 20 parents and 8 individuals with Down syndrome, were conducted virtually via videoconferencing to obtain participants' views of health indicators. Focus group moderators employed some modifications for individuals with Down syndrome, including simplified language and use of graphics.Transcripts were coded using a hybrid inductive/deductive framework and thematically analysed using the Framework Method. Results: We describe lessons learned in conducting virtual focus groups of individuals with Down syndrome and their parents. Individuals with Down syndrome could describe their views of health indicators and identified many of the same topics as their parents. Both groups discussed physical, mental, and social health components. However, people with Down syndrome gave a more restricted range of examples, but with different nuances than parents. Conclusion: Partcipants discussed physical, social, and mental well-being components of health in Down syndrome. Interviewing individuals with Down syndrome in virtual focus groups with appropriate modifications added important self-report health information.
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