Long-Term Tocilizumab Treatment Shows Sustained Relapse Reduction in MOGAD

Long-term treatment with the interleukin-6 receptor inhibitor tocilizumab (TCZ) was associated with sustained reductions in relapse activity and improvements in disability outcomes among patients with myelin oligodendrocyte glycoprotein antibody–associated disease (MOGAD), according to a recent longitudinal analysis.¹

Published in Neurology: Neuroimmunology & Neuroinflammation, the study—led by first author Francesco Capecchi and colleagues—adds to growing real-world evidence supporting IL-6 pathway inhibition in a disease where maintenance treatment strategies remain largely off-label and variably effective.

Study Design and Patient Population

The single-center study included 16 adult patients with MOGAD treated with TCZ for at least 6 months, with a median treatment duration of 2.5 years (range, 0.5–10.0).1 Patients had a mean age of 49.4 years, and 43.8% were female, with optic neuritis (81.3%) and myelitis (43.8%) representing the most common clinical phenotypes.

TCZ was used as first-line maintenance therapy in 62.5% of patients, while the remainder had previously received therapies such as rituximab or other immunosuppressants but switched due to ongoing disease activity or safety concerns.

Significant Reductions in Relapse Activity and Disability

Treatment with TCZ resulted in a statistically significant reduction in relapse activity, with median annualized relapse rate (ARR) decreasing from 0.75 (IQR, 0.5–1.0) in the 24 months prior to treatment to 0 (IQR, 0–0) in the first 12 months after initiation (95% CI, 0.4–1.0; p = 0.005). Only 2 of 16 patients (12.5%) experienced relapse while on therapy, with most disease activity occurring prior to TCZ initiation.

Led by senior author Marina Herwerth, MD, a physician in the neurology department at the University Hospital Zürich, these reductions in relapse burden were accompanied by modest but significant improvements in disability and visual outcomes. Median Expanded Disability Status Scale (EDSS) scores decreased from 2.8 at baseline to 2.3 at 12 months (p = 0.046), while median visual acuity improved from 0.5 (IQR, 0.3–0.9) prior to treatment to 0.9 (IQR, 0.5–1.0; p = 0.0003) at last follow-up.

Biomarker and Imaging Stability

Among patients with available serologic data, MOG-IgG titers decreased in 76.9% (10 of 13) within the first year of treatment. Radiologic outcomes were similarly stable, with no new lesions observed during follow-up and most patients demonstrating reduction or resolution of existing abnormalities.

As the authors noted, “Our long-term single-center experience suggests notable therapeutic effectiveness, as reflected by a marked and sustained decrease in ARR, with stabilization or improvement across clinical and paraclinical measures.”¹

Safety Profile and Long-Term Tolerability

Tocilizumab was generally well tolerated, with 81.3% of patients remaining on treatment at the time of analysis. Infectious adverse events occurred in 25% of patients, including 3 serious infections requiring hospitalization and 1 fatal case in an older patient with comorbidities.

Laboratory abnormalities were common but manageable. Among patients without baseline dyslipidemia, 75% developed new lipid abnormalities, though these were typically mild. Elevated liver enzymes occurred in 25% of patients without prior abnormalities and were transient. Subcutaneous administration was adopted in 31.3% of patients during follow-up, with all remaining relapse-free after switching, suggesting potential advantages for long-term adherence and patient convenience.

The authors added, “TCZ is generally well tolerated, but vigilance for infectious complications, especially in older multimorbid patients, remains critical throughout therapy.”¹

Clinical Context

These findings support IL-6 receptor inhibition as a mechanistically grounded and potentially effective approach in MOGAD, a condition in which existing therapies have shown inconsistent relapse prevention. While limited by its retrospective design and small sample size, the study provides further rationale for prospective trials to better define the role of TCZ and other IL-6–targeting agents in clinical practice.

REFERENCE
1. Capecchi F, Graure M, Yasaroglu S, et al. Long-term treatment with interleukin-6 receptor inhibitor tocilizumab in MOGAD. Neurol Neuroimmunol Neuroinflamm. 2026;2(1):e000049. doi:10.1212/WN9.0000000000000049