Pediatric Optic Neuritis—Building Foundations for a Randomized Clinical Trial

Abstract

Optic neuritis is a well-studied condition in adults. 1 It remains a rare and poorly understood disease in children, with limited information on causation, natural history, visual outcomes, associated n e u r o l o g i c a l d i s e a s e s , response to treatment, and prognosis. This is similar to the delay in the initial description of pediatric multiple sclerosis by Pierre Marie, MD, in 1893, occurring 15 years after the description of multiple sclerosis in adults by Jean-Martin Charcot, MD. Current concepts in pediatric optic neuritis (PON) are sometimes ambiguous or even conflicting, because published studies have generally been small and retrospective. 2 In response to this important need to better understand PON, an alliance between the Pediatric E y e D i s e a s e I n v e s t i g a t o r G r o u p a n d t h e N e u r o -Ophthalmology Research Disease Investigator Consortium planned an ambitious, prospective pilot observational study of PON. 3 In this issue of JAMA Ophthalmology, the Writing Committee for the Pediatric Eye Disease Investigator Group 4 reports the results of a multicenter observational prospective study in a pediatric population after an initial demyelinating optic neuritis attack. The main results of this large collaborative effort may seem disappointing, given that only 44 participants were enrolled from the 23 specialized centers, which is less than half of the planned target of 100 study participants. This difficulty in recruitment may have implications for the ability to recruit for a randomized clinical trial. Notwithstanding these enrollment difficulties, the current study provides valuable insights for future study designs. It provides important prospectively collected clinical information, suggesting that recruitment criteria may need to be relaxed to allow for more than 2 weeks from the time of vision loss to enrollment. It also provides realistic estimates for the time frame necessary for sufficient enrollment in a study.Unsurprisingly, both high-and low-contrast visual acuity in patients with PON was poor at baseline. Both improved in most patients within 6 months of symptom onset, but the sample size was insufficient to establish baseline prognostic factors for vision recovery. The factors associated with worse high-contrast visual acuity at presenta-