Abstract Details

Pediatric onset NMOSD is rare, representing only 3% of total cases. Like adults, presenting features include optic neuritis, myelitis, and brain syndromes. Three agents are now approved for treatment of adult NMOSD including eculizumab. There has been no published use of eculizumab in pediatric NMOSD patients.

Case report of a 4-year-old girl with a severe manifestation of AQP-4 NMOSD who had a refractory disease course despite aggressive immunotherapies including autologous hematopoietic stem cell transplant (AHSCT), who has been relapse free since starting eculizumab.

Case report featuring the clinical course of a pediatric patient with AQP-4 NMOSD who received eculizumab.

A 2-year-old girl presented with arm weakness and abnormal gait was found to be positive for AQP-4 IgG. She has had 14 relapses in the past 3 years including myelitis, optic neuritis, and parenchymal brain lesions. Relapses continued despite treatment with rituximab, mycophenolate mofetil, tocilizumab, chronic oral prednisone, and AHSCT, including 5 attacks in the 6 months prior to initiating eculizumab. The decision was made to start eculizumab given the severity of her disease and the likelihood of accumulating disability.

The patient received meningococcal vaccines and was placed on penicillin prophylaxis. She was initiated on eculizumab, 600 mg IV weekly (approximately 30 mg/kg), which was well tolerated. She has since remained relapse free for 4 months.

AQP-4 NMOSD has received increasing attention in recent years due to the development of FDA approved therapies to induce remission in adults. For pediatric NMOSD, the current standard of care are non-approved treatments including rituximab and mycophenolate mofetil. Due to the highly refractory nature of this patient’s disease, she was initiated on eculizumab. Thus far, she has been relapse free with good tolerability and no adverse events. The long-term safety and efficacy of eculizumab for children with AQP-4 NMOSD warrants further investigation.