Response to treatment in NMOSD: the Australasian experience

Laura Clarke; Wajih Bukhari; Cullen M O'Gorman; Elham Khalilidehkordi; Simon Arnett; Mark Woodhall; Kerri M Prain; John D E Parratt; Michael H Barnett; Mark P Marriott; Pamela A McCombe; Ian Sutton; Mike Boggild; Wallace Brownlee; William M Carroll; Suzanne Hodgkinson; Richard A L Macdonell; Deborah F Mason; Jennifer Pereira; Mark Slee; Chandi Das; Andrew P D Henderson; Allan G Kermode; Jeannette Lechner-Scott; ANZ NMO Collaboration; Patrick Waters; Jing Sun; Simon A Broadley

doi:10.1016/j.msard.2021.103408

Response to treatment in NMOSD: the Australasian experience

Mult Scler Relat Disord. 2022 Feb:58:103408. doi: 10.1016/j.msard.2021.103408. Epub 2021 Nov 21.

Authors

Laura Clarke¹, Wajih Bukhari², Cullen M O'Gorman³, Elham Khalilidehkordi⁴, Simon Arnett⁵, Mark Woodhall⁶, Kerri M Prain⁷, John D E Parratt⁸, Michael H Barnett⁹, Mark P Marriott¹⁰, Pamela A McCombe¹¹, Ian Sutton¹², Mike Boggild¹³, Wallace Brownlee¹⁴, William M Carroll¹⁵, Suzanne Hodgkinson¹⁶, Richard A L Macdonell¹⁷, Deborah F Mason¹⁸, Jennifer Pereira¹⁹, Mark Slee²⁰, Chandi Das²¹, Andrew P D Henderson²², Allan G Kermode²³, Jeannette Lechner-Scott²⁴; ANZ NMO Collaboration; Patrick Waters²⁵, Jing Sun²⁶, Simon A Broadley²⁷

Affiliations

¹ Menzies Health Institute Queensland, Gold Coast Campus, Griffith University QLD 4222, Australia; Department of Neurology Princess Alexandra Hospital, Woolloongabba QLD 4102, Australia.
² Menzies Health Institute Queensland, Gold Coast Campus, Griffith University QLD 4222, Australia; St Vincent's Hospital Melbourne, Fitzroy VIC 3065, AustraliA.
³ Menzies Health Institute Queensland, Gold Coast Campus, Griffith University QLD 4222, Australia; Department of Neurology Princess Alexandra Hospital, Woolloongabba QLD 4102, Australia; Department of Neurology, Mater Hospital Brisbane, South Brisbane QLD, 4101, Australia.
⁴ Menzies Health Institute Queensland, Gold Coast Campus, Griffith University QLD 4222, Australia; Department of Neurology, Royal Brisbane and Women's Hospital, Herston QLD 4029, Australia.
⁵ Menzies Health Institute Queensland, Gold Coast Campus, Griffith University QLD 4222, Australia; Department of Neurology, Gold Coast University Hospital, Southport QLD 4215, Australia.
⁶ Nuffield Department of Clinical Neurosciences, John Radcliffe Infirmary, University of Oxford, Oxford OX3 9DU, UK.
⁷ Department of Immunology, Pathology Queensland, Royal Brisbane and Women's Hospital, Herston QLD 4006, Australia.
⁸ Sydney Medical School, Royal Prince Alfred Hospital, University of Sydney, Camperdown NSW 2006, Australia.
⁹ Brain and Mind Research Institute, University of Sydney, Camperdown NSW 2006, Australia.
¹⁰ Melbourne Brain Centre, Royal Melbourne Hospital, University of Melbourne, Parkville VIC 3052, Australia.
¹¹ Department of Neurology, Royal Brisbane and Women's Hospital, Herston QLD 4029, Australia; Centre for Clinical Research, Royal Brisbane and Women's Hospital, University of Queensland, Herston QLD 4029, AustraliA.
¹² Department of Neurology, St Vincent's Hospital, Darlinghurst NSW 2010, Australia.
¹³ Department of Neurology, Townsville Hospital, Douglas QLD 4814, Australia.
¹⁴ Department of Neurology, Auckland City Hospital, Grafton 1023, New Zealand; Institute of Neurology, University College London, Queen Square, London WC1N 3BG, UK.
¹⁵ Centre for Neuromuscular and Neurological Disorders, Perron Institute for Neurological and Translational Science, Queen Elizabeth II Medical Centre, University of Western Australia, Nedlands WA 6009, AustraliA.
¹⁶ South Western Sydney Medical School, Liverpool Hospital, University of New South Wales, Liverpool NSW 2170, Australia.
¹⁷ Department of Neurology, Austin Health, Heidelberg VIC 3084, Australia.
¹⁸ Department of Neurology, Christchurch Hospital, Christchurch 8140, New Zealand.
¹⁹ Department of Neurology, Auckland City Hospital, Grafton 1023, New Zealand.
²⁰ Flinders Medical Centre, Flinders University, Bedford Park SA 5042, Australia.
²¹ Department of Neurology, Canberra Hospital, Garran ACT 2605, Australia.
²² Department of Neurology, Westmead Hospital, Westmead NSW 2145, Australia.
²³ Centre for Neuromuscular and Neurological Disorders, Perron Institute for Neurological and Translational Science, Queen Elizabeth II Medical Centre, University of Western Australia, Nedlands WA 6009, AustraliA; Institute for Immunology and Infectious Disease, Murdoch University, Murdoch WA 6150, AustraliA.
²⁴ Hunter Medical Research Institute, University of Newcastle, New Lambton Heights NSW 2305, AustralIA.
²⁵ Department of Neurology, Gold Coast University Hospital, Southport QLD 4215, Australia.
²⁶ Menzies Health Institute Queensland, Gold Coast Campus, Griffith University QLD 4222, Australia.
²⁷ Menzies Health Institute Queensland, Gold Coast Campus, Griffith University QLD 4222, Australia; Department of Neurology, Gold Coast University Hospital, Southport QLD 4215, Australia. Electronic address: simon.broadley@griffith.edu.au.

PMID: 35216788
DOI: 10.1016/j.msard.2021.103408

Abstract

Background: Neuromyelitis optica spectrum disorder (NMOSD) is associated with significant morbidity and mortality. Several therapies have been recommended for NMOSD and more recently clinical trials have demonstrated efficacy for three monoclonal antibody therapies. We present a retrospective observational study of treatment response in NMOSD.

Methods: This was a retrospective, unblinded, observational study of treatment efficacy for rituximab and traditional immunosuppressive therapy in patients with AQP4 antibody positive NMOSD. Treatment efficacy was assessed using annualised relapse rates (ARR), time to first relapse and expanded disability status scale (EDSS) scores.

Results: Complete relapse and treatment data were available for 43/68 (63%) of AQP4 antibody positive NMOSD cases covering 74 episodes of treatment. In a time to first relapse analysis rituximab showed a risk ratio of 0.23 (95% CI 0.08 - 0.65) when compared with no treatment and there was a non-significant reduction in ARR of 35% compared to pre-treatment. β-interferon (p = 0.0002) and cyclophosphamide (p = 0.0034) were associated with an increased ARR compared to pre-treatment. Rituximab (median 4.0 [range 0.0 - 7.0]; p = 0.042) and traditional immunosuppressive therapy (median 4.0 [range 0.0 - 8.0]; p = 0.016) were associated with a lower final EDSS compared to β-interferon (median 6.0 [range 4.0 - 7.5]).

Conclusions: These data provide additional support for the use of rituximab in preference to traditional immunosuppressive agents and MS disease modifying therapies as first line treatment of NMOSD.

Keywords: Aquaporin; Autoimmune disease; Clinical features; Multiple sclerosis; Neuromyelitis optica.

Publication types

Observational Study

MeSH terms

Aquaporin 4
Humans
Immunosuppressive Agents / therapeutic use
Neuromyelitis Optica* / drug therapy
Retrospective Studies
Rituximab / therapeutic use

Substances

Aquaporin 4
Immunosuppressive Agents
Rituximab