Eculizumab Reduces Relapse Risk in Neuromyelitis Optica Spectrum Disorder

The following article is part of conference coverage from the 8th Joint American Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) and European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) MSVirtual2020 event. Neurology Advisor‘s staff will be reporting breaking news associated with research conducted by leading experts in neurology. .

The terminal complement inhibitor eculizumab reduced the risk of relapse in patients with neuromyelitis optica spectrum disorder (NMOSD), regardless of disability burden, prior immunosuppressant therapy (IST) use, relapse history, or time since diagnosis, according to study results presented at the 8th Joint American Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) and European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) MSVirtual2020 event, held September 11-13, 2020.

In NMOSD, antibodies to the aquaporin-4 (AQP4) water channel reportedly trigger the complement cascade implicated in neuronal injury. Eculizumab is the first treatment approved for NMOSD patients who are AQP4 immunoglobulin G-positive. Using data from the PREVENT trial (ClinicalTrials.gov Identifier: NCT01892345), study investigators sought to determine if eculizumab’s benefits in reducing relapse risk in NMOSD are associated with disability burden, prior IST use, relapse history, or time since diagnosis.

In PREVENT, participants were given a maintenance dose of eculizumab (1200 mg/2 weeks) or a placebo, with a stable-dose concomitant IST (except mitoxantrone and rituximab) permitted. The trial was not powered for subgroup analyses. Post hoc descriptive analyses performed included subgroups defined by baseline expanded disability status scale (EDSS) score, prior IST use, total number of historical relapses, and time since NMOSD diagnosis.

The proportions of adjudicated relapse among participants were lower with eculizumab vs placebo. The proportions for eculizumab and placebo were, respectively, 0/14 vs 3/6 for baseline EDSS scores less than or equal to 2.0 and 3/82 vs 17/41 for baseline EDSS scores greater than or equal to 2.5 to less than or equal to 7.0; 0/15 vs 2/5 for no prior IST use (with the exception of corticosteroids alone), and 3/81 vs 18/42 for prior IST use; 1/39 vs 10/24 for 2 to 4 historical relapses and 2/57 vs 10/23 for greater than or equal to 5 historical relapses; 2/31 vs 6/12 for less than 1 year following diagnosis and 1/65 vs 14/35 for greater than or equal to 1 year following diagnosis. The reductions in relapse risk were statistically significant and consistent in all subgroups.

Study investigators concluded, “[t]he data from this post hoc subgroup analysis suggest that eculizumab reduced relapse risk in PREVENT compared with placebo, regardless of time since NMOSD diagnosis, relapse history, disability burden or prior IST use.”

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Reference

Fujihara K, Berthele A, Kim H, et al. Benefit of eculizumab for a broad range of patients with aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder: findings from PREVENT. Presented at: 8th Joint American Committee for Treatment and Research in Multiple Sclerosis and European Committee for Treatment and Research in Multiple Sclerosis MSVirtual2020 event; September 11-13, 2020. Abstract P0692.