Emerging Targeted Therapies for Neuromyelitis Optica Spectrum Disorders

doi:10.1007/s40259-020-00460-9

Review

. 2021 Jan;35(1):7-17.

doi: 10.1007/s40259-020-00460-9. Epub 2020 Dec 10.

Emerging Targeted Therapies for Neuromyelitis Optica Spectrum Disorders

Cristina Valencia-Sanchez¹, Dean M Wingerchuk²

Affiliations

¹ Department of Neurology, Mayo Clinic, Rochester, MN, USA.
² Department of Neurology, Mayo Clinic, 13400 East Shea Boulevard, Scottsdale, AZ, 85259, USA. wingerchuk.dean@mayo.edu.

PMID: 33301078
DOI: 10.1007/s40259-020-00460-9

Review

Emerging Targeted Therapies for Neuromyelitis Optica Spectrum Disorders

Cristina Valencia-Sanchez et al. BioDrugs. 2021 Jan.

. 2021 Jan;35(1):7-17.

doi: 10.1007/s40259-020-00460-9. Epub 2020 Dec 10.

Authors

Cristina Valencia-Sanchez¹, Dean M Wingerchuk²

Affiliations

¹ Department of Neurology, Mayo Clinic, Rochester, MN, USA.
² Department of Neurology, Mayo Clinic, 13400 East Shea Boulevard, Scottsdale, AZ, 85259, USA. wingerchuk.dean@mayo.edu.

PMID: 33301078
DOI: 10.1007/s40259-020-00460-9

Abstract

Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune, inflammatory disorder of the central nervous system that typically presents with recurrent episodes of optic neuritis, longitudinally extensive myelitis, brainstem, diencephalic, and cerebral syndromes. Up to 80% of NMOSD patients have a circulating pathogenic autoantibody that targets the water channel aquaporin-4 (AQP4-IgG). The discovery of AQP4-IgG transformed our understanding of the pathogenesis of the disease and its possible treatment targets. Monoclonal antibodies targeting terminal complement (eculizumab), CD19 (inebilizumab), and the interleukin-6 receptor (satralizumab) have demonstrated efficacy in NMOSD attack prevention in recent phase 3 trials and have gained subsequent regulatory approval in the USA and other countries. We aim to review the evidence supporting the efficacy of these new drugs.

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References

1. Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG. The spectrum of neuromyelitis optica. Lancet Neurol. 2007;6(9):805–15. https://doi.org/10.1016/s1474-4422(07)70216-8 . - DOI - PubMed
1. Wingerchuk DM, Banwell B, Bennett JL, Cabre P, Carroll W, Chitnis T, et al. International consensus diagnostic criteria for neuromyelitis optica spectrum disorders. Neurology. 2015;85(2):177–89. https://doi.org/10.1212/wnl.0000000000001729 . - DOI - PubMed - PMC
1. Kleiter I, Gahlen A, Borisow N, Fischer K, Wernecke KD, Wegner B, et al. Neuromyelitis optica: evaluation of 871 attacks and 1,153 treatment courses. Ann Neurol. 2016;79(2):206–16. https://doi.org/10.1002/ana.24554 . - DOI - PubMed
1. Marrie RA, Gryba C. The incidence and prevalence of neuromyelitis optica: a systematic review. Int J MS Care. 2013;15(3):113–8. https://doi.org/10.7224/1537-2073.2012-048 . - DOI - PubMed - PMC
1. Flanagan EP, Cabre P, Weinshenker BG, Sauver JS, Jacobson DJ, Majed M, et al. Epidemiology of aquaporin-4 autoimmunity and neuromyelitis optica spectrum. Ann Neurol. 2016;79(5):775–83. https://doi.org/10.1002/ana.24617 . - DOI - PubMed - PMC

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[1] Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG. The spectrum of neuromyelitis optica. Lancet Neurol. 2007;6(9):805–15. https://doi.org/10.1016/s1474-4422(07)70216-8 . - DOI - PubMed

[2] Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG. The spectrum of neuromyelitis optica. Lancet Neurol. 2007;6(9):805–15. https://doi.org/10.1016/s1474-4422(07)70216-8 . - DOI - PubMed

[3] Wingerchuk DM, Banwell B, Bennett JL, Cabre P, Carroll W, Chitnis T, et al. International consensus diagnostic criteria for neuromyelitis optica spectrum disorders. Neurology. 2015;85(2):177–89. https://doi.org/10.1212/wnl.0000000000001729 . - DOI - PubMed - PMC

[4] Wingerchuk DM, Banwell B, Bennett JL, Cabre P, Carroll W, Chitnis T, et al. International consensus diagnostic criteria for neuromyelitis optica spectrum disorders. Neurology. 2015;85(2):177–89. https://doi.org/10.1212/wnl.0000000000001729 . - DOI - PubMed - PMC

[5] Kleiter I, Gahlen A, Borisow N, Fischer K, Wernecke KD, Wegner B, et al. Neuromyelitis optica: evaluation of 871 attacks and 1,153 treatment courses. Ann Neurol. 2016;79(2):206–16. https://doi.org/10.1002/ana.24554 . - DOI - PubMed

[6] Kleiter I, Gahlen A, Borisow N, Fischer K, Wernecke KD, Wegner B, et al. Neuromyelitis optica: evaluation of 871 attacks and 1,153 treatment courses. Ann Neurol. 2016;79(2):206–16. https://doi.org/10.1002/ana.24554 . - DOI - PubMed

[7] Marrie RA, Gryba C. The incidence and prevalence of neuromyelitis optica: a systematic review. Int J MS Care. 2013;15(3):113–8. https://doi.org/10.7224/1537-2073.2012-048 . - DOI - PubMed - PMC

[8] Marrie RA, Gryba C. The incidence and prevalence of neuromyelitis optica: a systematic review. Int J MS Care. 2013;15(3):113–8. https://doi.org/10.7224/1537-2073.2012-048 . - DOI - PubMed - PMC

[9] Flanagan EP, Cabre P, Weinshenker BG, Sauver JS, Jacobson DJ, Majed M, et al. Epidemiology of aquaporin-4 autoimmunity and neuromyelitis optica spectrum. Ann Neurol. 2016;79(5):775–83. https://doi.org/10.1002/ana.24617 . - DOI - PubMed - PMC

[10] Flanagan EP, Cabre P, Weinshenker BG, Sauver JS, Jacobson DJ, Majed M, et al. Epidemiology of aquaporin-4 autoimmunity and neuromyelitis optica spectrum. Ann Neurol. 2016;79(5):775–83. https://doi.org/10.1002/ana.24617 . - DOI - PubMed - PMC

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Emerging Targeted Therapies for Neuromyelitis Optica Spectrum Disorders

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