Controversies in the management of neuromyelitis optica spectrum disorder

ABSTRACT

Introduction: Neuromyelitis optica spectrum disorders (NMOSD) are autoimmune diseases of the central nervous system mainly involving the optic nerves and spinal cord. Many advances have been made in understanding the immunopathology of NMOSD and related clinical classification, nevertheless, open issues in management and effective therapeutic approaches still remain.

Areas covered: In this article, the authors reviewed and discussed the scientific evidence in pathogenesis and pharmacological therapy of NMOSD addressing the more recent advances in new biological treatment option and therapeutic strategy that may help to improve management of this condition.

Expert opinion: Despite current immunopathogenic evidence, NMOSD management represents a challenge due to the poor-validated diagnostic, prognostic and therapeutic biomarkers. A tailored approach is mandatory to improve the management of the different disease clinical settings.

KEYWORDS:

• Neuromyelitis optica spectrum disorders (NMOSD)
• diagnostic criteria
• immunopathogenesis
• aquaporin-4 immunoglobulin G (AQP4-IgG)
• myelin oligodendrocyte glycoprotein immunoglobulin G (MOG-IgG)
• standard treatment
• emerging treatment
• management

Article highlights

• Neuromyelitis optica spectrum disorders (NMOSD) is a rare and clinically aggressive disease of the central nervous system characterized by a wider spectrum of clinical manifestations.

• The antibodies against water channel protein aquaporin 4 (AQP4IgG) have a key role in pathogenesis and diagnosis of NMOSD.

• New immunopathologic evidence suggests novel potential biomarkers

• Conventional immunosuppressive and B-cell depletion therapies, specifically Rituximab, represent the standard treatment

• Emerging treatments targeting specific elements in immunologic pathways such as complement pathway, interleukin-6 signaling, and Th17 cells have been investigated through phase III clinical trials and may represent the gold standard therapy.

In this review, we provide an overview of NMOSD management focusing on present and future open challenges.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

A peer reviewer on this manuscript receives royalties from RSR Ltd, Oxford University, Hospices Civil de Lyon, and MVZ Labor PD Dr. Volkmann und Kollegen GbR for a patent of NMO-IgG as a diagnostic test for NMO and related disorders, served on adjudication committee for clinical trials in NMO being conducted by MedImmune and Alexion, and consulted for Chugai regarding a clinical trial for NMO. A second peer reviewer on this manuscript is a site principal investigator in a randomized placebo-controlled clinical trial of Inebilizumab (A CD19 inhibitor) in neuromyelitis optica spectrum disorders funded by MedImmune/Viela Bio. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.

Additional information

Funding

This paper was not funded.