Pediatric MOGAD: Brain lesions and optic neuritis outcomes - Children's National

In a new retrospective study published in Multiple Sclerosis and Related Disorders, researchers analyzed pediatric patients with myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) to determine whether the presence of brain lesions affected clinical outcomes in those presenting with optic neuritis (ON). By comparing patients with ON and brain lesions to those with ON without brain lesions, the investigators found that brain lesions were often asymptomatic and were not associated with additional clinical disability.

The big picture

The study retrospectively looked at 32 pediatric patients with MOGAD being followed by Children's National Hospital and Johns Hopkins Hospital between August 2010 and July 2024. Of these, 18 presented with ON without brain lesions and 14 with ON and brain lesions. Overall, 69% of patients were female and the median age at symptom onset was 12 years old, with a younger onset among those experiencing acute disseminated encephalomyelitis. Across the cohort, a total of 35 ON episodes were analyzed.

Features of ON, including severity of visual impairment, were largely consistent between groups, although individuals with concurrent brain lesions more frequently exhibited additional neurologic symptoms. Outcomes were favorable overall, with substantial recovery noted by one month and minimal disability at three months and beyond. Follow-up imaging demonstrated improvement in most brain lesions over time, suggesting that neither their presence nor persistence reliably predicts long-term disability or a more severe ON presentation.

The hold up in the field

"MOGAD is a rare disorder that has only been commercially testable since 2017, so we are still learning about its clinical phenotypes and long-term outcomes," said Vivien Xie, MD, pediatric neuroimmunology fellow at Children's National and lead author of the study. "In pediatrics, it can be especially difficult to conduct research on rare diseases because large cohort numbers are needed to draw meaningful conclusions."

Moving the field forward

MOGAD encompasses a range of clinical phenotypes, and it remains unclear whether these phenotypes carry distinct prognostic implications in children.

"This study advances our understanding of prognosis in MOGAD and supports a more individualized approach to monitoring and treatment, while also helping clinicians more confidently counsel patients and families about the implications of brain lesions identified on initial MRI," said Dr. Xie.

Read the study, Significance of brain lesions associated with optic neuritis in pediatric myelin oligodendrocyte glycoprotein antibody-associated disease, in the journal Multiple Sclerosis and Related Disorders.

Additional authors from Children's National include: Claire Har, Kyle Spagnolo, DO, Ilana Kahn, MD, Leigh Sepeta, PhD, Alexandra B. Kornbluh, MD.