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 recommended by the EBMT and ectrims 1.
Overview of NMO Treatment
The management of NMO involves both acute and long-term strategies. For acute attacks, high-dose intravenous methylprednisolone is often used, followed by plasma exchange if necessary. However, the focus has shifted towards long-term prevention of relapses using immunosuppressive therapies.
Long-term Prevention Strategies
Several medications are used for long-term prevention, including:
- Rituximab, which targets B cells
- Eculizumab, an anti-IL-6 receptor monoclonal antibody
- Inebilizumab, satralizumab, and other complement-inhibiting monoclonal antibodies
- Azathioprine and mycophenolate mofetil, which are immunosuppressive drugs
These treatments aim to reduce the frequency and severity of relapses, thereby improving the quality of life for patients with NMO.
Role of HSCT in NMO
Autologous haematopoietic stem cell transplantation (AHSCT) and allogeneic HSCT are considered clinical options for NMO that is refractory to conventional treatment, as endorsed by the EBMT 1. However, the role of HSCT in NMO has been explored in only a few studies, with mixed outcomes. A registry analysis by the EBMT ADWP reported progression-free survival at years 3-5 to be 48%, but 81% of patients experienced a relapse at a median of 7 months after AHSCT.
Recent Recommendations
The most recent study from 2025 recommends AHSCT and allogeneic HSCT as clinical options for the treatment of NMO that is refractory to conventional treatment 1. This study highlights the importance of considering HSCT as a treatment option for patients with NMO who have not responded to other therapies.
Key Considerations
When managing NMO, it is essential to consider the following:
- The disease is typically relapsing, with each attack potentially causing cumulative neurological disability
- Treatment should be continued indefinitely in most cases
- Supportive care, including physical therapy, management of neuropathic pain, and addressing bladder dysfunction, is crucial for comprehensive NMO management
By prioritizing the use of highly effective pharmacological treatments and considering HSCT as a treatment option for refractory cases, patients with NMO can experience improved outcomes and quality of life.
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