Advances in the therapeutic landscape for neuromyelitis optica spectrum disorder

Neuromyelitis optica spectrum disorder (NMOSD) is a rare, relapsing autoimmune disorder of the central nervous system, characterized by inflammatory attacks affecting the optic nerves, spinal cord and brain. In most cases, pathogenic antibodies against aquaporin-4 (AQP4-IgG) are detectable. These antibodies target astrocytes, triggering complement activation and the release of proinflammatory cytokines such as interleukin-6 (IL-6), culminating in an astrocytopathy and neurological disability.

In this review, Medical Subject Heading (MeSH) and related entry terms were used to search English literature in PubMed and ClinicalTrial.gov databases, respectively. MeSH included but were not limited to Neuromyelitis optica (NMO), Neuromyelitis Optica Spectrum Disorders (NMOSD), pathogenesis, complement, immune cells, B-cell, T-cell, cytokines, and therapy.

Over the past decade, significant strides in our understanding of NMOSD immunopathogenesis have led to the development of targeted therapies that have revolutionized patient outcomes and survival. This review outlines the clinical features of NMOSD and examines current and emerging therapeutic strategies in the context of disease biology. It also addresses ongoing challenges, including optimization of acute attack management, strategies for treatment de-escalation, and prospects for achieving immune tolerance.