Development and Validation of the Pediatric Hypersomnolence Survey.

BACKGROUND AND OBJECTIVES: Narcolepsy and idiopathic hypersomnia usually begin in early adolescence, but diagnostic delays ranging from 5 to 10 years are common, affecting disease burden. To improve early identification of these treatable conditions, we developed and validated the Pediatric Hypersomnolence Survey (PHS). METHODS: Content was developed through literature review, patient focus groups, interviews with experts in the field, and field testing. We then validated the 14-item self-reported survey across 3 hospitals and web recruitment from patient groups. In the validation phase, we recruited a total of 331 participants (patients with narcolepsy type 1 [n = 64], narcolepsy type 2 [n = 34], idiopathic hypersomnia [n = 36], and other sleep disorders [n = 97] and healthy controls [n = 100], ages 8-18 years) to complete the survey. We assessed a range of psychometric properties, including discriminant diagnostic validity for CNS disorders of hypersomnolence using receiver operating characteristic curve analysis and reliability across a 1-week period. RESULTS: Confirmatory factor analysis indicated a 4-domain solution with good reliability expressed by satisfactory omega values. Across groups, the PHS total score showed appropriate positive correlations with other validated surveys of sleepiness (r = 0.65-0.78, p < 0.001) and negative correlations with multiple sleep latency test measures (mean sleep latency: r = -0.27, p = 0.006; number of sleep-onset REM periods: r = 0.26, p = 0.007). Compared to controls and patients with other sleep disorders, the area under the curve for participants with narcolepsy or idiopathic hypersomnia was 0.87 (standard error 0.02, 95% CI 0.83-0.91) with high sensitivity (81.3, 95% CI 73.7%-87.5%) and specificity (81.2%, 95 CI 75.1%-86.4%). Test-retest reliability was r = 0.87. DISCUSSION: The PHS is a valid and reliable tool for clinicians to identify pediatric patients with narcolepsy and idiopathic hypersomnia. Implemented in clinical practice, the PHS will potentially decrease diagnostic delays and time to treatment, ultimately reducing disease burden for these debilitating conditions. CLASSIFICATION OF EVIDENCE: This study provides Class III evidence that the PHS accurately identifies patients with central disorders of hypersomnolence.

Remote Assessment of Pediatric Patients with Daytime Sleepiness and Healthy Controls: A Pilot Study of Feasibility and Reliability.

We assessed the reliability of cognitive testing for children and adolescents ages 8 to 19 years of age with narcolepsy or subjective daytime sleepiness compared to healthy controls. Forty-six participants took part in the study (n = 18 narcolepsy type 1, n = 6 subjective daytime sleepiness, and n = 22 healthy controls). Participants completed verbal (vocabulary testing) and non-verbal intelligence quotient (IQ) tasks (block design, matrix reasoning) from the Wechsler Abbreviated Scale of Intelligence- Second Edition (WASI-II) in-person or remotely through a HIPAA compliant telehealth platform with conditions counterbalanced. We found that vocabulary T-scores showed good reliability with intraclass correlation coefficient (ICC) of 0.76 (95% CI: 0.64, 0.85) between remote and in-person testing conditions. Matrix Reasoning T-scores showed moderate reliability (ICC 0.69, 95% CI: 0.68, 0.90) and Block Design T-scores was poor between testing conditions. Overall, the results of this pilot study support the feasibility and reliability of verbal and non-verbal IQ scores collected by telehealth.

Remote Assessment of Cognition in Kids and Adolescents with Daytime Sleepiness: A pilot study of feasibility and reliability.

In this pilot study, we assessed the reliability of cognitive testing for kids and adolescents ages 8-19 years of age with narcolepsy or subjective daytime sleepiness compared to healthy controls. Forty-six participants took part in the study (n=18 with narcolepsy type 1, n=6 with subjective daytime sleepiness, and n= 22 healthy controls recruited from the community). Participants completed verbal (vocabulary testing) and non-verbal intelligence quotient (IQ) tasks (block design, matrix reasoning) from the Weschler Abbreviated Scale of Intelligence-Second Edition (WASI-II) in-person or remotely in their home through a HIPAA compliant telehealth web platform with conditions counterbalanced. We found that vocabulary T-scores showed good reliability with intraclass correlation coefficient (ICC) of 0.76 (95% CI: 0.64, 0.85) between remote and in-person testing conditions. Matrix Reasoning T-scores showed moderate reliability (ICC 0.69, 95% CI: 0.68, 0.90) and Block Design T-scores was poor between testing conditions. Bland-Altman plots showed outliers on vocabulary and matrix reasoning tasks performed better on remote assessments. Overall, the results of this pilot study support the feasibility and reliability of verbal and non-verbal IQ scores collected by telehealth. Use of telehealth to collect verbal and non-verbal IQ scores may offer a means to acquire cognitive data for pediatric sleep research through the COVID-19 pandemic and beyond.