Abstract

Background: Neuromyelitis optica spectrum disorder (NMOSD), an autoimmune inflammatory disorder of the central nervous system, often leads to vision loss or paralysis. This meta-analysis focused on the assessment of the monoclonal antibody therapy in NMOSD and compared different targets of monoclonal antibodies with each other in terms of efficacy and safety outcomes.Method: We searched through the databases of MEDLINE, EMBASE, Central Register of Controlled Trials (CENTRAL), and clinicaltrials.gov for randomized controlled trials (RCTs) evaluating monoclonal antibody therapy in NMOSD up to April 2020.Results: We identified seven randomized controlled trials (RCTs), including 775 patients (monoclonal antibody group, n = 485 and placebo group, n = 290). Monoclonal antibody therapy decreased relapse risk (RR 0.33, 95% CI 0.21–0.52, P < 0.00001), annualized relapse rate (ARR) (mean −0.28, 95% CI −0.35−0.20, P < 0.00001), expanded disability status scale score (EDSS) (mean −0.19, 95% CI −0.32−0.07, P = 0.002) and serious adverse events (RR 0.78, 95% CI 0.61–1.00, P = 0.05). However, we did not observe any significant difference in terms of adverse events or mortality. Further, the subgroup analysis demonstrated that the anti-complement protein C5 monoclonal antibody (eculizumab) might have a lower relapse risk (RR 0.07, 95% CI 0.02–0.23, P < 0.0001) in the AQP4 seropositive patients, and anti-interleukin-6 receptor monoclonal antibodies (satralizumab and tocilizumab) showed decreased EDSS score (mean −0.17, 95% CI −0.31−0.02, P = 0.02) more effectively than other monoclonal antibodies.Conclusions: Monoclonal antibodies were effective and safe in NMOSD. Different targets of monoclonal antibodies might have their own advantages.

Highlights

  • Neuromyelitis optica spectrum disorder (NMOSD) is a relapsing inflammatory autoimmune disease of the central nervous system whose symptoms are associated with optic nerve, spinal cord, brain stem, and cerebrum injury

  • NMOSD patients had a very low mortality (3/775) in 7 included randomized controlled trial (RCT) and no statistically significant difference was observed in mortality from the monoclonal antibody group to the placebo group (RR 1.18, 95% confidence interval (CI) 0.15–9.47, P = 0.87; Figure 3C)

  • From the perspective of the expanded disability status scale (EDSS) score change, we detected that anti-interleukin-6 receptor monoclonal antibodies exhibited significantly a better performance to improve functional recovery than other monoclonal antibodies

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Summary

Introduction

Neuromyelitis optica spectrum disorder (NMOSD) is a relapsing inflammatory autoimmune disease of the central nervous system whose symptoms are associated with optic nerve, spinal cord, brain stem, and cerebrum injury. To reduce relapse risk immunosuppressive drugs such as azathioprine (AZA), mycophenolate mofetil (MMF), and monoclonal antibodies like rituximab are frequently used in clinical practices [8,9,10,11]. Neuromyelitis optica spectrum disorder (NMOSD), an autoimmune inflammatory disorder of the central nervous system, often leads to vision loss or paralysis. This meta-analysis focused on the assessment of the monoclonal antibody therapy in NMOSD and compared different targets of monoclonal antibodies with each other in terms of efficacy and safety outcomes

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