Nature and Science of Sleep (Aug 2017)
Assessing narcolepsy with cataplexy in children and adolescents: development of a cataplexy diary and the ESS-CHAD
Abstract
Y Grace Wang,1 Khadra Benmedjahed,2 Jérémy Lambert,2 Christopher J Evans,3 Steve Hwang,3 Jed Black,1,4 Murray W Johns5 1Clinical Development, Jazz Pharmaceuticals, Palo Alto, CA, USA; 2Patient-Centered Outcomes, Mapi, Lyon, France; 3Endpoint Outcomes, Boston, MA, USA; 4Sleep Medicine, Stanford Sleep Medicine Center, Redwood City, CA, USA; 5Epworth Sleep Centre, East Melbourne, VIC, Australia 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 ESS-CHAD 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. Keywords: narcolepsy, cataplexy, sleepiness, assessment, children, adolescents
