Pediatric optic neuritis (ON) is a rare disease that has not been well characterized. The pediatric ON prospective outcomes study (PON1) was the first prospective study aiming to evaluate visual acuity (VA) outcomes, including VA, recurrence risk, and final diagnosis 2-years post-enrollment DESIGN: Non-randomized observational study at 23 pediatric ophthalmology or neuro-ophthalmology clinics in US and Canada.
28 (64%) of 44 children initially enrolled in PON1 (age 3<16 years) who completed their 2-year study visit.
Treatment at investigator discretion.
Age-normal monocular high contrast VA (HCVA). Secondary outcomes included low contrast VA (LCVA), neuroimaging findings, and final diagnoses.
28 participants completed the 2-year outcome with a median enrollment age of 10.3 years (range 5-15); 46% were female and 68% had unilateral ON at presentation. Final 2-year diagnoses included isolated ON (n=11, 40%), myelin oligodendrocyte glycoprotein (MOG)-associated demyelination (n=8, 29%), multiple sclerosis (MS) (n=4,14%), neuromyelitis optica spectrum disease (NMOSD) (n=3,11%), and acute disseminated encephalomyelitis (n=2, 7%). Two (7%; 95% CI=1-24%) participants had subsequent recurrent ON (plus one participant who did not complete the 2-year visit); all had MS. Two other participants (7%) had a new episode in their unaffected eye. Mean presenting HCVA was 0.81 logMAR (∼20/125), improving to 0.14 logMAR (∼20/25) at 6 months, 0.12 logMAR (∼20/25) at 1 year, and 0.11 logMAR (20/25) at 2 years (95% CI=-0.08-0.3 (20/20-20/40)). Twenty-four (79%) had age-normal VA at 2 years (95% CI=60-90%);21 (66%) had 20/20 vision or better. The six participants without age-normal VA had 2-year diagnoses of NMOSD (n=2 participants, 3 eyes), MS (n=2 participants, 2 eyes), and isolated ON (n=2 participants, 3 eyes). Mean presenting LCVA was 1.45 logMAR (∼20/500), improving to 0.78 logMAR (∼20/125) at 6 months, 0.69 logMAR (∼20/100+1) at 1 year, and 0.68 logMAR (∼20/100) at 2 years (95% CI=0.48-0.88 (20/50-20/150)).
Despite poor VA at presentation, most children had marked improvement in VA by 6 months which was maintained over two years. Associated neurologic autoimmune diagnoses were common. Additional episodes of ON occurred in 5 (18%) of the participants (3 relapses and 2 new episodes).

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