0658 Validity and Reliability of the Pediatric Narcolepsy Patient-Reported Outcomes Scale (PN-PROS)
Bibliographic record
Abstract
Abstract Introduction There are currently no validated, patient-reported outcome measures for pediatric narcolepsy that assess disease burden, determine treatment efficacy, and guide future drug development. To ensure optimal clinical management of pediatric narcolepsy, we developed the Pediatric Narcolepsy Patient-Reported Outcomes Scale (PN-PROS) through literature review, content expert interviews, patient focus groups, and cognitive testing with patients (ages 9-17 years) and their parents. The aim of this study is to provide interim data from our multi-site validation and reliability study of the PN-PROS. Methods We performed field testing for validity and reliability of the PN-PROs in pediatric narcolepsy patients (9-17 years) with a comparator group of pediatric obstructive sleep apnea patients. We recruited participants from Boston Children’s Hospital, Stanford Medical Center, Toronto Hospital for Sick Children (SickKids), and Geisinger Medical Center, as well as from narcolepsy patient advocacy meetings and websites. Participants completed the PN-PROS, Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD), Peds QL, and PROMIS Life Satisfaction using the REDCap data capture platform. Participants completed the PN-PROS item bank 1 week later for test-retest reliability. Results 83 pediatric patients with narcolepsy (mean age=15(2) years, 52.3% female, 26% non-Caucasian) and 60 pediatric patients with OSA (mean age=13.2(2.6), 46.3% female, 22% non-Caucasian) have completed all study measures. Discriminant Validity: Participants with narcolepsy reported a higher PN-PROS mean total score than participants with OSA [narcolepsy=126.9 (28.6), OSA=95 (31.2), p< 0.001]; results retain significance controlling for age, race and gender [group main effect: F=36.6, p< 0.001]. Content Validity: For participants with narcolepsy, the PN-PROS total score was significantly correlated with the ESS-CHAD (r=0.64, p< 0.001), Peds QL (r=-0.84, p< 0.001), and PROMIS Life Satisfaction (r=-0.55, p< 0.001). Reliability: Internal consistent was strong for both participants with narcolepsy (Cronbach alpha=0.94) and OSA (Cronbach alpha=0.93). Test-retest reliability was high for both participants with narcolepsy (interclass coefficient=0.94) and OSA (interclass coefficient=0.93). Conclusion Interim results suggest the PN-PROS is a valid and reliable measure for the evaluation of symptom frequency and burden for pediatric patients with narcolepsy. Data collection is ongoing, utilizing additional sites in other regions of the United States to ensure generalizability of our findings. Support (if any) American Academy of Sleep Medicine
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.006 | 0.014 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.001 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.001 |
| Research integrity | 0.000 | 0.001 |
| Insufficient payload (model declined to judge) | 0.003 | 0.001 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".