Highlights from

EAN 2020

European Academy of Neurology

Virtual 23 - 26 May 2020

Eculizumab in NMOSD: the PREVENT study

In the phase 3, randomised, double-blind PREVENT study, eculizumab was effective in aquaporin-4 immunoglobulin G-positive neuromyelitis optica spectrum disorder (NMOSD) in different regions of the world, despite significant differences in standard of care [1]. Long-term results from the PREVENT study’s open-label extension (OLE) supported the efficacy of eculizumab monotherapy in reducing relapse risk [2].

The PREVENT study assessed the efficacy and safety of eculizumab in adult patient populations in Europe, the Americas, and Asia-Pacific. Patients were randomised to eculizumab or placebo. Stable doses of concomitant immunosuppressive therapy (IST) were allowed. IST use at baseline was much lower in Europe than in the other 2 regions, so that a large proportion of European participants in the placebo group had no medication at all. Compared with placebo, eculizumab significantly reduced relapse risk, relapse-related hospitalisation, and rates of acute relapse treatment with intravenous methylprednisolone across regions [1].

These results suggested a treatment effect of eculizumab monotherapy that is consistent with the overall population. A post-hoc analysis of long-term results from the OLE of the PREVENT study confirmed the efficacy of eculizumab monotherapy in reducing relapse risk [2]. Relapse-related disability outcomes and healthcare resource utilisation were reported in a subgroup of 34 patients (24%) who received eculizumab monotherapy or placebo without concomitant IST use. The majority (95%) experienced stability or improvement in measures of disability. In the prespecified subgroup analysis, adjudicated relapses occurred in 0/21 patients receiving eculizumab monotherapy and 7/13 (53.8%) receiving placebo (P<0.0001). Fewer patients in the eculizumab group had disability progression. In the placebo group, 6/13 patients (46.2%) were hospitalised for adjudicated relapses and received treatment. In the eculizumab and in the placebo group, Expanded Disability Status Scale (EDSS) scores worsened in 5/13 (38.5%) patients, respectively (P=0.151). Hauser Ambulation Index score worsened in 1/21 (4.8%) and 4/13 (30.8%), respectively (P=0.205).

Keywords: Neuromyelitis Optica; Eculizumab; PREVENT

  1. Oreja-Guevara C, et al. Abstract O4009, EAN 2020.
  2. Pittock SJ, et al. Abstract LB90, EAN 2020.

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