Assessing narcolepsy with cataplexy in children and adolescents: development of a cataplexy diary and the ESS-CHAD

Objective: The aim of this study was to qualitatively evaluate concepts for incorporation into a daily diary to capture cataplexy frequency and to assess the content validity of the Epworth Sleepiness Scale for Children and Adolescents (ESS- CHAD) in pediatric patients with narcolepsy. Patients and methods: Face- to- face concept elicitation and cognitive interviews were conducted with children (7- 9 years; n=13) and adolescents (10- 17 years; n=16) who have narcolepsy with cataplexy, and their parents/caregivers. Results: Similarities and differences were noted between narcolepsy concepts described by children and their parents/caregivers, suggesting some different but complementary perspectives; parents may not recognize cataplexy symptoms/triggers as well as children, but parents have greater recognition of the circumstances of falling asleep. Cataplexy diary modifications included changes in definitions and examples of cataplexy, using child- friendly terminology, adding a quantitative question to determine daily frequency, and standardizing the questionnaire for evening administration with self- completion by the child. Modifications were made to ESSCHAD for child- friendly wording and to ensure that items reflect activities (eating, watching TV/ video) and environments (school, bus/car transport) in which children are likely to participate. Two ESS- CHAD versions were proposed: one with a 1- month recall period, for general use, and the other with a recall period of since your last study visit,for research, which could be shorter or longer than 1 month (as short as 1 week). Conclusion: The cataplexy diary and ESS- CHAD were modified for the assessment of children and adolescents. Further psychometric validation is recommended. These measures are being used in a Phase III, placebo- controlled clinical trial of sodium oxybate in children and adolescents with narcolepsy.