From the Guidelines
The treatment of neuromyelitis optica (NMO) should prioritize the use of highly effective pharmacological treatments, including B cell-depleting, anti-IL-6 receptor, and complement-inhibiting monoclonal antibodies, as they have been shown to effectively suppress disease activity in recent clinical trials 1.
Overview of NMO Treatment
The management of NMO involves both acute attack management and long-term immunosuppressive therapy. For acute attacks, high-dose intravenous methylprednisolone is often used, followed by oral prednisone taper. If response is inadequate, plasma exchange should be initiated promptly.
Long-term Prevention
For long-term prevention, rituximab is commonly used, with alternative options including eculizumab, inebilizumab, or satralizumab. These treatments work by targeting different aspects of the immune system, particularly the antibodies against aquaporin-4 water channels that characterize the disease.
Autologous Haematopoietic Stem Cell Transplantation (AHSCT)
AHSCT is endorsed by the EBMT as a clinical option for the treatment of NMO that is refractory to conventional treatment, with a progression-free survival of 90% at year 5 in a prospective open-label cohort study 1. However, the role of HSCT in NMO has been explored in only a few studies, and outcomes have been mixed.
Key Considerations
- Long-term therapy is essential as NMO is a relapsing condition where attacks can cause permanent disability.
- Symptomatic management of pain, spasticity, bladder dysfunction, and neuropathic symptoms is also important for comprehensive care.
- The use of older immunosuppressants like azathioprine, mycophenolate mofetil, or methotrexate may be considered when newer therapies are unavailable.
Recent Recommendations
The most recent study published in 2025 in Nature Reviews Neurology 1 provides recommendations from ectrims and the ebmt on the use of AHSCT for the treatment of NMO, highlighting its potential as a clinical option for refractory cases.
Disease Activity Suppression
The use of highly effective pharmacological treatments, including B cell-depleting, anti-IL-6 receptor, and complement-inhibiting monoclonal antibodies, has been shown to effectively suppress disease activity in recent clinical trials 1, making them a priority in the treatment of NMO.
From the Research
Treatment Options for Neuromyelitis Optica
- Azathioprine (AZA) and rituximab (RIT) are two commonly used treatments for neuromyelitis optica spectrum disorder (NMOSD) 2, 3, 4, 5
- RIT has been shown to be more effective than AZA in reducing annualized relapse rate (ARR) and Expanded Disability Status Scale (EDSS) scores 2, 4, 5
- Mycophenolate mofetil (MMF) is also used as a treatment option for NMOSD, but RIT has been found to be superior to MMF in reducing the risk of relapse and improving disability 4, 5
Acute Treatment
- High-dose intravenous corticosteroid pulse and plasmapheresis are commonly used to treat acute NMOSD attacks 3, 6
- Early and accurate diagnosis of NMOSD is crucial for preventing disability associated with the disease 6
Long-term Immunotherapy
- AZA, MMF, and RIT are used as first-line, long-term immunotherapies for NMOSD 3, 6, 4, 5
- RIT has been found to be effective in reducing the risk of relapse and improving disability in patients with NMOSD, with a lower incidence of adverse events compared to AZA 4, 5
Emerging Therapies
- New therapy strategies using monoclonal antibodies like RIT have been tested in NMOSD, with positive results in open-label studies 3
- Emerging therapies based on the current understanding of the disease immunopathogenesis are being developed, including targeted efforts to develop novel, disease-specific treatments 6