Long-Term Treatment With Interleukin-6 Receptor Inhibitor Tocilizumab in Myelin Oligodendrocyte Glycoprotein Antibody–Associated Disease
Data about efficacy and safety of disease-modifying therapeutic options in myelin oligoden- drocyte glycoprotein antibody–associated disease (MOGAD) are scarce. Interleukin-6 sig- naling has been suggested to be involved in the pathogenesis of MOGAD. The aim of this study was to evaluate the effectiveness and safety of the interleukin-6 receptor inhibitor tocilizumab (TCZ) in MOGAD. In this longitudinal, retrospective study, we included patients diagnosed with MOGAD at a single tertiary referral center who received treatment with TCZ for at least 6 months. The annualized relapse rate (ARR), the Expanded Disability Status Scale (EDSS) scores, imaging findings, autoantibody titers, and adverse events were evaluated before and after initiation of TCZ. The assessment of side effects, such as dyslipidemia, impaired liver function, and infectious complications, was based on anamnestic, clinical, and laboratory data. Sixteen patients (mean age 49.4 years, range 28–71; 43.8% female) with a median observation period of 2.5 years on TCZ treatment (range 0.5–10.0) were enrolled. The median ARR significantly decreased from 0.75 (interquartile range [IQR] 0.5–1.0) at 24 months before TCZ initiation to 0 (IQR 0-0) at 12 months after TCZ initiation (95% CI 0.4–1.0, p = 0.005). The EDSS score improved from 2.8 (IQR 2.0–3.5) at baseline to 2.3 (IQR 1.1–3.0) at 12 months after starting TCZ (95% CI 0.0–1.5, p = 0.046). The median visual acuity improved significantly from 0.5 (IQR 0.3–0.9) before initiation of TCZ to 0.9 (IQR 0.5–1.0; p = 0.0003) at the last follow-up. In the first year after starting TCZ treatment, MOG-IgG titers decreased in 10 of 13 patients (76.9%). No patient showed radiologic progression under TCZ. Infectious adverse events requiring medical treatment occurred in 4 of 16 patients (25%). Side effects such as dyslipidemia and impaired liver function were frequent but rarely required the interruption of TCZ. In 5 patients (31.3%), the route of administration of TCZ was switched to subcutaneous injections over the disease course. TCZ shows promise as a potentially effective treatment in MOGAD, and its subcutaneous administration may improve long-term therapy adherence. However, the retrospective design and small cohort size limit generalizability. Future prospective randomized trials are needed to determine its definitive efficacy.
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