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Abstract
Neuromyelitis optica and neuromyelitis optica spectrum disorder (NMO/NMOSD) is a rare but clinically aggressive demyelinating disease of the central nervous system (CNS) caused by antibodies against water channel protein aquaporin 4 (AQP4) in the astrocytic foot processes. Patients typically present with optic neuritis (ON) or longitudinally extensive transverse myelitis (LETM). The majority of patients with NMOSD show good response to treatment with steroids and plasmapheresis in the acute setting; however, 90 % of patients will eventually have clinical relapses and accrue permanent disability. Currently, immune modulation is the mainstay of maintenance therapy with anti CD-20 (rituximab, Rituxan™) having collectively the strongest evidence to support its use and mycophenolate mofetil having comparable reductions in absolute relapse rate (ARR) and expanded disability status scale (EDSS) scores. Azathioprine, mitoxantrone, and methotrexate also have retrospective case series data that demonstrate reduction in ARR and stabilization of EDSS but with higher relapse rates and exposure to greater risk of treatment toxicities. Excitingly, multiple novel therapies are under clinical study for patients who are refractory to these first-line therapies including monoclonal antibodies targeting interleukin-6 (IL-6), CD19, CD20, complement, and neutrophil elastase inhibitors which may provide additional options for patients with severe clinical presentations. Importantly, no randomized clinical trials have been published to date comparing clinical outcomes of different maintenance therapies in NMOSD. Several trials are currently underway, and results will help guide future management decisions as current evidence is from many small, retrospective case series and cohort studies with many potential confounds.
View details for DOI 10.1007/s11940-015-0378-x
View details for PubMedID 26433388
View details for PubMedCentralID PMC4592697