Long-term Outcomes in Childhood-onset Aquaporin-4 positive Neuromyelitis Optica Spectrum Disorder: Data from a Prospective Cohort
Sujatha Manjunathan1, Naveen Sankhyan1, Renu Suthar1, Arushi Gahlot Saini1, Jitendra Sahu1, Sameer Vyas2, Arun Bansal1, Amit Rawat1
1Pediatrics, 2Radiodiagnosis and Imaging, Postgraduate Institute of Medical Education and Research
Objective:

To assess long-term outcomes in Childhood-onset Aquaporin-4-positive Neuromyelitis Optica Spectrum Disorder

Background:

Neuromyelitis Optica Spectrum Disorder (NMOSD) encompasses autoimmune demyelinating disorders of the central nervous system with predilection towards optic nerves and the spinal cord. Unlike adults, Aquaporin-4(AQP-4) positive NMOSD is exceedingly rare in children. Prospective data on the clinical presentation, frequency of relapses, response to therapy, and residual deficits are scarce in childhood-onset AQP-4-positive NMOSD.

Design/Methods:

This nested case series was drawn from a prospective cohort study on childhood demyelinating disorders initiated after the Institute’s ethical committee approval in 2018. All children diagnosed and followed up during the seven years with AQP-4-positive NMOSD were analyzed for associated autoimmunity, frequency of relapses, response to therapy, residual deficits, and outcomes. 

Results:

A total of 12 children were diagnosed with AQP-4-positive NMOSD during the study period. There were nine girls and three boys (M:F: 1:3). The median age at onset was 8.5 years. The first attack was transverse myelitis in 7, optic neuritis in 2, optic neuritis with transverse myelitis in 1, and brainstem syndrome in 2. Overall, 6 had optic neuritis, and 9 had transverse myelitis. A total of 7 children had relapses, and the total relapses were 16. Three had another associated autoimmune disease (2 - Juvenile Dermatomyositis and 1 - Anti-NMDAR encephalitis). Over a median follow-up of 25 months, the Annualized Relapse Rate was 0.2, with a median Expanded Disability Status Scale (EDSS) score of 3.5. Rituximab was used as a relapse prevention therapy in 10 children, and 9/10 remained relapse-free after initiation of Rituximab. One child who defaulted on treatment died.

Conclusions:

AQP-4-positive NMOSD in children is a rare but severe disease with residual disabilities. Associated autoimmunity is seen in children with AQP-4-positive NMOSD. In this study, Rituximab appeared to be efficacious in preventing relapses in most children. 

10.1212/WNL.0000000000212018
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