REVIEW PAPER
 
KEYWORDS
TOPICS
ABSTRACT
Introduction and objective:
Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune demyelinating disease of the central nervous system, distinct from multiple sclerosis. Its pathogenesis is primarily associated with anti-AQP4-IgG antibodies, though some patients present with anti-MOG-IgG. The most common symptoms include optic neuritis and longitudinal extensive transverse myelitis (LETM) often leading to blindness and tetra- or paraparesis. This review summarizes current and emerging treatment strategies for NMOSD, including therapies used in acute attacks, long-term immunosuppression, and targeted biological agents.

Review methods:
The paper is based on a review of current literature and clinical trial data carried out in March 2025 using electronic databases: PubMed, Scopus and Web of Science. It discusses conventional immunosuppressants (e.g., azathioprine, mycophenolate mofetil, rituximab), targeted therapies (satralizumab, eculizumab, inebilizumab), and the latest Polish drug programme (B.138.FM). Differential diagnostics of NMOSD and radiological imaging of patients is shown.

Brief description of the state of knowledge:
Biologic therapies show significant effectiveness in relapse prevention, especially in AQP4-IgG-positive patients. Satralizumab and inebilizumab reduce relapse rates and disability progression, with favourable safety profiles. Eculizumab provides rapid and sustained complement inhibition, offering high efficacy. Satralizumab is now reimbursed in Poland for eligible patients.

Summary:
Modern, targeted therapies for NMOSD greatly improve patient outcomes and quality of life. Accurate and timely diagnosis, access to appropriate treatment, and individualized therapeutic approaches remain key to reducing long-term disability in NMOSD.
REFERENCES (38)
1.
Kümpfel T, Giglhuber K, Aktas O, et al.; Neuromyelitis Optica Study Group (NEMOS). 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. 2024 Jan;271(1):141–76. https://doi.org/10.1007/s00415....
 
2.
Carnero Contentti E, Correale J. Neuromyelitis optica spectrum disorders: from pathophysiology to therapeutic strategies. J Neuroinflammation. 2021 Sep 16;18(1):208. https://doi.org/10.1186/s12974....
 
3.
Zara P, Dinoto A, Carta S, et al. Non-demyelinating disorders mimicking and misdiagnosed as NMOSD: a literature review. Eur J Neurol. 2023 Oct;30(10):3367–76. https://doi.org/10.1111/ene.15....
 
4.
George M, Gantioque R. Neuromyelitis optica spectrum disorder: An early diagnosis to prevent blindness and paraplegia. Nurse Pract. 2023;48(6):104654. https://doi.org/10.1016/j.nurp....
 
5.
Hor JY, Asgari N, Nakashima I, et al. Epidemiology of neuromyelitis optica spectrum disorder and its prevalence and incidence worldwide. Front Neurol. 2020 Jun 26;11:501. https://doi.org/10.3389/fneur.....
 
6.
Giglhuber K, Berthele A. Adverse events in NMOSD therapy. Int J Mol Sci. 2022 Apr 9;23(8):4154. https://doi.org/10.3390/ijms23....
 
7.
Chan KH, Lee CY. Treatment of neuromyelitis optica spectrum disorders. Int J Mol Sci. 2021 Aug 11;22(16):8638. https://doi.org/10.3390/ijms22....
 
8.
Shumway CL, Patel BC, Tripathy K, et al. Neuromyelitis optica spectrum disorder (NMOSD) [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan 8. https://www.ncbi.nlm.nih.gov/b... (access: 2025.03.23).
 
9.
Rodríguez-Acevedo B, Rovira A, Vidal-Jordana A, et al. Neuritis óptica: etiopatogenia, diagnóstico, pronóstico y manejo [Optic neuritis: aetiopathogenesis, diagnosis, prognosis and management]. Rev Neurol. 2022 Feb 1;74(3):93–104. https://doi.org/10.33588/rn.74....
 
10.
Beck RW, Cleary PA, Anderson MM Jr, et al. A randomized, controlled trial of corticosteroids in the treatment of acute optic neuritis. N Engl J Med. 1992 Feb 27;326(9):581–8. https://doi.org/10.1056/NEJM19....
 
11.
Çakar A, Ulusoy C, Gündüz T, et al. Clinical features of the patients with neuromyelitis optica spectrum disorder. Noro Psikiyatr Ars. 2019 Aug 26;58(1):21–5. https://doi.org/10.29399/npa.2....
 
12.
Wingerchuk DM, Banwell B, Bennett JL, et al. International consensus diagnostic criteria for neuromyelitis optica spectrum disorders. Neurology. 2015 Jul 14;85(2):177–89. https://doi.org/10.1212/WNL.00....
 
13.
Dinoto A, Sechi E, Flanagan EP, et al. Serum and cerebrospinal fluid biomarkers in neuromyelitis optica spectrum disorder and myelin oligodendrocyte glycoprotein associated disease. Front Neurol. 2022 Mar 23;13:866824. https://doi.org/10.3389/fneur.....
 
14.
Kitley J, Woodhall M, Leite MI, et al. Aquaporin-4 antibody isoform binding specificities do not explain clinical variations in NMO. Neurol Neuroimmunol Neuroinflamm. 2015 Jun 18;2(4):e121. https://doi.org/10.1212/NXI.00....
 
15.
Jarius S, Aktas O, Ayzenberg I, et al.; Neuromyelitis Optica Study Group (NEMOS). Update on the diagnosis and treatment of neuromyelitis optica spectrum disorders (NMOSD) – revised recommendations of the Neuromyelitis Optica Study Group (NEMOS). Part I: Diagnosis and differential diagnosis. J Neurol. 2023 Jul;270(7):3341–68. https://doi.org/10.1007/s00415....
 
16.
Cacciaguerra L, Sechi E, Rocca MA, et al. Neuroimaging features in inflammatory myelopathies: a review. Front Neurol. 2022 Oct 18;13:993645. https://doi.org/10.3389/fneur.....
 
17.
Cao G, Duan Y, Zhang N, et al. Brain MRI characteristics in neuromyelitis optica spectrum disorders: a large multi-center retrospective study in China. Mult Scler Relat Disord. 2020 Nov;46:102475. https://doi.org/10.1016/j.msar....
 
18.
Kosiyakul P, Songwisit S, Ungprasert P, et al. Effect of plasma exchange in neuromyelitis optica spectrum disorder: a systematic review and meta-analysis. Ann Clin Transl Neurol. 2020 Nov;7(11):2094–102. https://doi.org/10.1002/acn3.5....
 
19.
Li X, Tian DC, Fan M, et al. Intravenous immunoglobulin for acute attacks in neuromyelitis optica spectrum disorders (NMOSD). Mult Scler Relat Disord. 2020 Nov;46:102325. https://doi.org/10.1016/j.msar....
 
20.
Luo D, Wei R, Tian X, et al. Efficacy and safety of azathioprine for neuromyelitis optica spectrum disorders: a meta-analysis of real-world studies. Mult Scler Relat Disord. 2020 Nov;46:102484. https://doi.org/10.1016/j.msar....
 
21.
Akatani R, Chihara N, Koto S, et al. Efficacy and safety of mycophenolate mofetil for steroid reduction in neuromyelitis optica spectrum disorder: a prospective cohort study. Immunol Med. 2024;47(2):85–92. https://doi.org/10.1080/257858....
 
22.
Dong GY, Meng YH, Xiao XJ. A meta-analysis on efficacy and safety of rituximab for neuromyelitis optica spectrum disorders. Medicine (Baltimore). 2022 Sep 9;101(36):e30347. https://doi.org/10.1097/MD.000....
 
23.
Yamamura T, Kleiter I, Fujihara K, et al. Trial of satralizumab in neuromyelitis optica spectrum disorder. N Engl J Med. 2019 Dec 5;381(23):2114–24. https://doi.org/10.1056/NEJMoa....
 
24.
Traboulsee A, Greenberg BM, Bennett JL, et al. Safety and efficacy of satralizumab monotherapy in neuromyelitis optica spectrum disorder: a randomised, double-blind, multicentre, placebo-controlled phase 3 trial. Lancet Neurol. 2020 May;19(5):402–12. https://doi.org/10.1016/S1474-....
 
25.
European Commission. Annex I: Summary of product characteristics – Enspryng (satralizumab). Brussels: European Commission; 2021 Jun 24 https://ec.europa.eu/health/do... (access: 2025.03.10).
 
26.
Ministry of Health. Summary of Product Characteristics: Enspryng (satralizumab). Warsaw: Ministry of Health; 2021. https://www.gov.pl/attachment/... (access: 2025.02.24).
 
27.
Chu YC, Huang TL. What’s new in neuromyelitis optica spectrum disorder treatment? Taiwan J Ophthalmol. 2022 Sep 1;12(3):249–63. https://doi.org/10.4103/2211-5....
 
28.
Pittock SJ, Fujihara K, Palace J, et al. Eculizumab monotherapy for NMOSD: data from PREVENT and its open-label extension. Mult Scler. 2022 Mar;28(3):480–6. https://doi.org/10.1177/135245....
 
29.
Wingerchuk DM, Fujihara K, Palace J, et al. Long-term safety and efficacy of eculizumab in aquaporin-4 IgG-positive NMOSD. Ann Neurol. 2021 Jun;89(6):1088–98. https://doi.org/10.1002/ana.26....
 
30.
Bennett JL, Aktas O, Rees WA, et al. Association between B-cell depletion and attack risk in neuromyelitis optica spectrum disorder: an exploratory analysis from N-MOmentum, a double-blind, randomised, placebo-controlled, multicentre phase 2/3 trial. EBioMedicine. 2022 Dec;86:104321. https://doi.org/10.1016/j.ebio....
 
31.
Aktas O, Hartung HP, Smith MA, et al. Serum neurofilament light chain levels at attack predict post-attack disability worsening and are mitigated by inebilizumab: analysis of four potential biomarkers in neuromyelitis optica spectrum disorder. J Neurol Neurosurg Psychiatry. 2023 Sep;94(9):757–68. https://doi.org/10.1136/jnnp-2....
 
32.
Bennett JL, Pittock SJ, Paul F, et al. B cell and aquaporin-4 antibody relationships with neuromyelitis optica spectrum disorder activity. Ann Clin Transl Neurol. 2024 Oct;11(10):2792–8. https://doi.org/10.1002/acn3.5....
 
33.
Cree BAC, Kim HJ, Weinshenker BG, et al. Safety and efficacy of inebilizumab for the treatment of neuromyelitis optica spectrum disorder: end-of-study results from the open-label period of the N-MOmentum trial. Lancet Neurol. 2024 May;23(5):403–14. https://doi.org/10.1016/S1474-....
 
34.
Zhang C, Zhang M, Qiu W, et al. Safety and efficacy of tocilizumab versus azathioprine in highly relapsing neuromyelitis optica spectrum disorder (TANGO): an open-label, multicentre, randomised, phase 2 trial. Lancet Neurol. 2020 May;19(5):391–401. https://doi.org/10.1016/S1474-....
 
35.
Elsone L, Townsend T, Mutch K, et al. Neuropathic pruritus (itch) in neuromyelitis optica. Mult Scler. 2013 Apr;19(4):475–9. https://doi.org/10.1177/135245....
 
36.
He Z, Ren M, Wang X, et al. Pruritus may be a common symptom related to neuromyelitis optica spectrum disorders. Mult Scler Relat Disord. 2017 Apr;13:1–3. https://doi.org/10.1016/j.msar....
 
37.
Liu J, Zhang X, Zhong Y, et al. The prevalence of depression, anxiety, and sleep disturbances in patients with neuromyelitis optica spectrum disorders (NMOSD): a systematic review and meta-analysis. Mult Scler Relat Disord. 2023 Nov;79:105007. https://doi.org/10.1016/j.msar....
 
38.
Yamamura T, Weinshenker B, Yeaman MR, et al. Long-term safety of satralizumab in neuromyelitis optica spectrum disorder (NMOSD) from SAkuraSky and SAkuraStar. Mult Scler Relat Disord. 2022 Oct;66:104025. https://doi.org/10.1016/j.msar....
 
eISSN:1898-7516
ISSN:1898-2395
Journals System - logo
Scroll to top