What is the treatment for neuromyelitis optica?

Treatment of Neuromyelitis Optica

Rituximab is the most effective treatment for neuromyelitis optica (NMO) and should be considered first-line therapy, particularly for patients with severe disease or those who have failed other immunosuppressive treatments. 1

Acute Treatment

First-Line Acute Treatment

1. High-dose intravenous methylprednisolone

   • 1g/day for 3-5 days 2
   • Should be initiated promptly, ideally within the first few hours of symptom onset 1
   • Follow with oral prednisone in tapering doses

2. Plasmapheresis (Plasma Exchange)

   • Indicated for steroid-refractory cases 1
   • Clinical improvement seen in approximately 79.2% of patients 1
   • Should be considered early, especially in severe attacks

Long-Term Immunosuppressive Therapy

First-Line Preventive Treatments

1. Rituximab (RTX)

   • Most effective option with superior relapse reduction compared to azathioprine 1, 3
   • 73% of poor responders to first-line immunosuppressants showed good response when switched to rituximab 4
   • Increasingly regarded as established therapy with long-term efficacy and acceptable safety profile 3

2. Azathioprine (AZA)

   • Traditional first-line option 5, 3
   • Less effective than rituximab, particularly in patients with:
     • History of severe attacks 4
     • Younger age at disease onset 4

3. Mycophenolate Mofetil (MMF)

   • Alternative first-line option 5, 3
   • Demonstrated significant decrease in mean EDSS scores 1
   • Similar limitations as azathioprine in high-risk patients 4

Second-Line Preventive Treatments

1. Cyclophosphamide

   • Particularly effective when combined with IV methylprednisolone 1
   • Consider for severe, refractory cases 2

2. Other options:

   • Methotrexate 3
   • Mitoxantrone 3
   • Tocilizumab (anti-IL6 receptor) - emerging therapy 3

Treatment Algorithm

1. For acute attacks:

   • Start IV methylprednisolone immediately (1g/day for 3-5 days)
   • If inadequate response within 3-5 days, initiate plasmapheresis
   • Follow with oral prednisone taper

2. For long-term prevention:

   • High-risk patients (history of severe attacks, younger age at onset):
     • Start with rituximab
   • Standard-risk patients:
     • Option 1: Rituximab
     • Option 2: Azathioprine or mycophenolate mofetil
   • Monitor closely for relapses during corticosteroid tapering

Important Considerations

• Early diagnosis and treatment are critical for preventing disability 6

• Relapses are common (50-60%) during corticosteroid dose reduction, highlighting the need for maintenance immunosuppressive therapy 1

• Poor prognostic factors include:

  • Extensive spinal cord MRI lesions
  • Reduced muscle strength or sphincter dysfunction at presentation
  • Presence of antiphospholipid antibodies
  • Delay (>2 weeks) in treatment initiation 1
  • Younger age at disease onset 4

• NMO is distinct from multiple sclerosis - MS treatments such as interferons may worsen NMO 5

Emerging Therapies

• Mesenchymal stem cell infusion has shown marked reduction in relapses in clinical trials 1
• Anti-complement therapies are being investigated based on the complement-mediated pathophysiology of NMO 3
• Anti-aquaporin-4 antibody biologicals are in development 3

Monitoring

• Regular clinical assessments for relapse
• MRI monitoring of optic nerves and spinal cord
• Testing for aquaporin-4 antibodies (AQP4-Ab/NMO-IgG) is essential for diagnosis and helps distinguish NMO from other autoimmune diseases 3