To examine health outcomes in terms of individual dimensions from the 5-dimension EuroQoL 3-level questionnaire (EQ-5D-3L): mobility, self-care, usual activities, pain/discomfort and anxiety/depression, for patients with neuromyelitis optica spectrum disorder (NMOSD) treated with the terminal complement inhibitor eculizumab.

Relapses in NMOSD can cause neurological deficits, resulting in significant negative health outcomes. In the phase 3, randomized, double-blind, placebo-controlled PREVENT study (NCT01892345) conducted in patients with aquaporin-4 immunoglobulin G (AQP4-IgG)-positive NMOSD, mean changes from baseline for the EQ-5D-3L visual analog scale score were 5.4 (standard deviation [SD], 18.53) for eculizumab and 0.6 (16.39) for placebo (p = 0.0309); corresponding changes for the EQ-5D-3L index score were 0.05 (0.179) and −0.04 (0.212), respectively (p = 0.0077).

Patients were randomized 2:1 to receive eculizumab (maintenance dose, 1200 mg/2 weeks) or placebo. At baseline and 4, 8, 12 and every 12 weeks thereafter until the end of the study, patients completed the EQ-5D-3L. For each dimension, the distribution of changes in patients reporting no, some or extreme problems between baseline and study end were analyzed post hoc using a randomization-based non-parametric analysis of covariance.

Of 143 patients randomized (eculizumab, n = 96; placebo, n = 47), the median age was 45.0 years and 90.9% were female. At baseline, most patients reported some or extreme problems in the EQ-5D-3L dimensions. These proportions were greatest for the dimension pain/discomfort (eculizumab, 81.25%; placebo, 82.98%) and least for self-care (eculizumab, 32.29%; placebo, 36.17%). Changes in the proportions reporting problems favored eculizumab over placebo for all dimensions. Differences were significant for usual activities (p = 0.0014), self-care (p = 0.0181) and pain/discomfort (p = 0.0359).

In patients with AQP4-IgG-positive NMOSD, eculizumab improved health outcomes compared with placebo, measured as changes in patients reporting problems in EQ-5D-3L dimensions.