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Current Treatment Landscape

AQP4-IgG ANTIBODY TREATMENT HAS 2 GOALS: TO SUPPRESS ACUTE INFLAMMATORY ATTACKS AND TO REDUCE RISK FOR RELAPSES1

When managing neuromyelitis optica spectrum disorder (NMOSD), approved and off-label treatments are used to suppress current attacks and reduce the risk for relapses.1-3 Corticosteroids are often used as first-line treatment to address acute inflammatory attacks, and immunosuppressants can be initiated concomitantly with the goal of reducing the risk for future relapses.1,2

AQP4-IgG, aquaporin-4 immunoglobulin G.
This is a hypothetical patient.

SOME CURRENT TREATMENT APPROACHES HAVE CRITICAL LIMITATIONS WHEN USED LONG TERM2,4,5

While off-label treatments can offer certain benefits, they may also have critical limitations when used long term.2,4,5 Treatments like immunosuppressants have not been studied in placebo-controlled trials.2 Therefore, the current treatment landscape is based largely on case studies, retrospective studies, and expert opinion.2

IMMUNOSUPPRESSANTS

Characteristics2
  • May decrease annualized relapse rate
  • Limited studies show improvements in Expanded Disability Status Scale (EDSS)
Long-term
Adverse Events2
  • Bone-marrow depletion
  • Diabetes
  • Leukopenia
  • Need for co-treatment with a corticosteroid
  • Psychiatric symptoms

CORTICOSTEROIDS

Characteristics4
  • Widely available
  • Extensive track record
  • Low cost
  • Immunosuppressive mechanism of action (MOA)
Long-term
Adverse Events2,4,5
  • Cardiovascular issues (hypertension, arrhythmias)
  • Cushing's syndrome
  • Electrolyte imbalances
  • Fungal and viral infections
  • Gastritis and peptic ulcers
  • Gastrointestinal issues
  • Increased risk of infections
  • Metabolic conditions (hyperglycemia, diabetes)
  • Myopathy
  • Neuropsychiatric symptoms
  • Ophthalmologic conditions
  • Osteoporosis
  • Thrombosis
  • Weight gain

CERTAIN CLINICAL CONCERNS DIMINISH PATIENT SATISFACTION6

In a study* on patient treatment experience, more than 50% of participants (51.8%) reported having concerns regarding their NMOSD treatment. Their concerns were mostly about on future effectiveness.

The majority of patients who changed treatments did so because of efficacy concerns, and 36% reported intolerable side effects.

*
Based on a cross-sectional study assessing the physical, emotional, and socioeconomic toll of NMOSD on quality of life (QOL) in 193 patients.

COULD RECENTLY APPROVED TREATMENTS OFFER NEW POSSIBILITIES FOR PATIENTS WITH NMOSD?

Several treatments have recently been FDA approved for NMOSD and continue to be developed and studied in clinical trials.7-9 These new treatment approaches help to reduce the risk of relapse and may offer more selective immune system inhibition.10,11

ATTACKS CAN BE RECURRENT AND MAY PREDICT FUTURE DISABILITY12,13

CYCLE OF ATTACKS

COMPLEMENT ACTIVATION IS AN IMPORTANT CAUSE OF ASTROCYTE DESTRUCTION IN NMOSD12,14-17

COMPLEMENT IN NMOSD
References: 1. Kessler RA, Mealy MA, Levy M. Treatment of neuromyelitis optica spectrum disorder: acute, preventive, and symptomatic. Curr Treat Options Neurol. 2016;18(1):2. 2. Borisow N, Mori M, Kuwabara S, Scheel M, Paul F. Diagnosis and treatment of NMO spectrum disorder and MOG-encephalomyelitis. Front Neurol. 2018;9:888. 3. Chan KH, Lee CY. Treatment of neuromyelitis optica spectrum disorders. Int J Mol Sci. 2021;22(16):8638. 4. Johnson S, Katyal N, Narula N, Govindarajan R. Adverse side effects associated with corticosteroid therapy: A study in 39 patients with generalized myasthenia gravis. Med Sci Monit. 2021;27:e933296. 5. Carroll WM, Fujihara K. Neuromyelitis optica. Curr Treat Options Neurol. 2010;12(3):244-255. 6. Beekman J, Keisler A, Pedraza O, et al. Neuromyelitis optica spectrum disorder: patient experience and quality of life. Neurol Neuroimmunol Neuroinflamm. 2019;6(4):e580. 7. FDA approves first treatment for neuromyelitis optica spectrum disorder, a rare autoimmune disease of the central nervous system. News release. US Food & Drug Administration. Published June 27, 2019. Accessed July 22, 2019. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-neuromyelitis-optica-spectrum-disorder-rare-autoimmune-disease-central 8. FDA approves treatment for rare disease affecting optic nerves, spinal cord. News release. US Food & Drug Administration. Published August 17, 2020. Accessed January 22, 2021. https://www.fda.gov/news-events/press-announcements/fda-approves-treatment-rare-disease-affecting-optic-nerves-spinal-cord 9. An efficacy and safety study of ravulizumab in adult participants with NMOSD. Clinicaltrials.gov. Published December 17, 2019. Updated August 9, 2023. Accessed January 22, 2024. https://clinicaltrials.gov/ct2/show/NCT04201262 10. SOLIRIS. Package insert. Alexion Pharmaceuticals, Inc.; 2020. 11. Enspryng. Package insert. Genentech, Inc.; 2022. 12. Jarius S, Ruprecht K, Wildemann B, et al. Contrasting disease patterns in seropositive and seronegative neuromyelitis optica: a multicentre study of 175 patients. J Neuroinflammation. 2012;9:14. 13. Kitley J, Leite MI, Nakashima I, et al. Prognostic factors and disease course in aquaporin-4 antibody-positive patients with neuromyelitis optica spectrum disorder from the United Kingdom and Japan. Brain. 2012;135(pt 6):1834-1849. 14. Piatek P, Domowicz M, Lewkowicz N, et al. C5a-preactivated neutrophils are critical for autoimmune-induced astrocyte dysregulation in neuromyelitis optica spectrum disorder. Front Immunol. 2018;9:1694. 15. Winkler A, Wrzos C, Haberl M, et al. Blood-brain barrier resealing in neuromyelitis optica occurs independently of astrocyte regeneration. J Clin Invest. 2021;131(5):e141694. 16. Chamberlain JL, Huda S, Whittam DH, Matiello M, Morgan BP, Jacob A. Role of complement and potential of complement inhibitors in myasthenia gravis and neuromyelitis optica spectrum disorders: a brief review. J Neurol. 2021;268(5):1643-1664. 17. Mealy MA, Kessler RA, Rimler Z, et al. Mortality in neuromyelitis optica is strongly associated with African ancestry. Neurol Neuroimmunol Neuroinflamm. 2018;5(4):e468.