Validation of a symptom-based questionnaire for pediatric CNS demyelinating diseases

Amy T. Waldman, Anusha K. Yeshokumar, Amy Lavery, Geraldine Liu, Stacy L. Pineles, Michael X. Repka, Laura Adang, Sona Narula, Grant T. Liu

Research output: Contribution to journalArticlepeer-review

2 Scopus citations


Purpose: Optic neuritis is a manifestation of numerous neuroinflammatory disorders. Recognition of current and prior symptoms may facilitate identification of an underlying multifocal neurologic disease. The purpose of this study was to determine whether a symptom-based questionnaire could inform clinical decision making by identifying children with visual complaints who may have a systemic demyelinating disorder. Methods: Children with visual changes from non-demyelinating disease were compared with patients with confirmed pediatric-onset multiple sclerosis (MS) or neuromyelitis optica spectrum disorder (NMOSD). Participants completed a 21-item questionnaire to capture their recent (<30 days) and remote (>30 days) symptoms of neurologic dysfunction. The questionnaire scores were compared using t tests, and the 95% confidence interval for each group was used to determine a threshold score suggesting demyelinating disease. Results: We enrolled 51 participants (30 females [59%]) with a mean age of 14.6 years (range, 4-21): 25 in the non-demyelinating disease group and 26 with MS/NMOSD. The mean questionnaire score for the non-demyelinating group was 5.0 points (95% CI, 3.3-6.9); for the MS/NMOSD group, 9.4 points (95% CI, 7.4-11.4) for the MS/NMOSD group (P < 0.002). Questionnaire results were dichotomized using a score of ≥7 as indicative of demyelinating disease, with 69% sensitivity and 72% specificity. An abbreviated questionnaire, using 8 questions that differed between groups, had a sensitivity of 65% and specificity of 92%. Conclusions: A symptom-based questionnaire is sensitive and specific for identifying children with CNS demyelinating disease and may be useful as a screening tool for children with vision complaints and possible demyelination.

Original languageEnglish (US)
Pages (from-to)157.e1-157.e7
JournalJournal of AAPOS
Issue number3
StatePublished - Jun 2019

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Ophthalmology


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