| Neuroimmunology | Rare Neurological Diseases  

Update on the diagnosis and treatment of neuromyelitis optica spectrum disorders (NMOSD)

The text provides an overview of neuromyelitis optica spectrum disorders (NMOSD), including its subtypes, diagnostic markers, treatment recommendations, and recent advancements in therapies, with a focus on differentiating NMOSD from multiple sclerosis and other related conditions.

The term 'neuromyelitis optica spectrum disorders' (NMOSD) encompasses aquaporin-4 immunoglobulin G (AQP4-IgG)-positive neuromyelitis optica (NMO) and related clinical syndromes without AQP4-IgG. Originally considered subvariants of multiple sclerosis (MS), NMOSD is now recognized as distinct in terms of immunopathogenesis, clinical presentation, treatment, and prognosis. Part 1 (1) of this series by the Neuromyelitis Optica Study Group (NEMOS), a Germany-wide network of NMO researchers and clinical neurologists, focuses on updated recommendations for diagnosing NMOSD, emphasizing differentiation from MS and myelin oligodendrocyte glycoprotein antibody-associated encephalomyelitis (MOGAD). Part 2 (2) covers treatment recommendations, including newly approved drugs and established options, for managing acute attacks and implementing preventive immunotherapies. AQP4-IgG antibodies are diagnostic markers, influencing disease pathogenicity. Recent advancements have led to new therapies like eculizumab, ravulizumab, inebilizumab, and satralizumab for AQP4-IgG-positive NMOSD. NEMOS provides insights on NMOSD treatments, emphasizing therapy management and immunotherapy use. Unmet needs and AQP4-IgG-negative NMOSD are also discussed. Recommendations were developed through a consensus method among experts at NEMOS meetings.

Key Points:

  1. The diagnosis of NMOSD should be made according to the 2015 International Panel for NMO Diagnosis (IPND) criteria
  2. Confirmation of positive AQP4-IgG with cell-based assay (CBA) is recommended, while for NMOSD with negative or unknown AQP4-IgG status, the typical MRI criteria must be met.
  3. Eculizumab, ravulizumab, inebilizumab, rituximab, and satralizumab have all demonstrated benefit in RCTs for AQP4-IgG-positive NMOSD, although there is a lack of efficient data from eculizumab, ravulizumab, inebilizumab, and satralizumab trials as a “first-line” therapy.
  4. Although there are no approved therapies for double-negative NMOSD, treatment recommendations for this group of patients are usually based on expert opinions, and include classical immunosuppressive therapies and rituximab.

References:

  1. Jarius, S., Aktas, O., Ayzenberg, I. et al. Update on the diagnosis and treatment of neuromyelits optica spectrum disorders (NMOSD) – revised recommendations of the Neuromyelitis Optica Study Group (NEMOS). Part I: Diagnosis and differential diagnosis. J Neurol 270, 3341–3368 (2023). doi.org/10.1007/s00415-023-11634-0
  2. Kümpfel, T., Giglhuber, K., Aktas, O. et al. Update on the diagnosis and treatment of neuromyelitis optica spectrum disorders (NMOSD) – revised recommendations of the Neuromyelitis Optica Study Group (NEMOS). Part II: Attack therapy and long-term management. J Neurol 271, 141–176 (2024). doi.org/10.1007/s00415-023-11910-z

Co-Author:
Dr. Matteo Gastaldi

Publish on behalf of the Scientific Panel on Neuroimmunology

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