ArticleJournal of neurology2026
Tocilizumab for severe immune checkpoint inhibitor myopathy-myocarditis: acute and long-term outcomes.
Article in Journal of neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
backgroundImmune checkpoint inhibitor (ICI)-myopathy-myocarditis is an immune-related adverse event, with high mortality despite first-line immunosuppression. Emerging data suggest upregulation of the interleukin-6 (IL-6) pathway in ICI-myopathy, raising interest in IL-6 receptor blockade as escalation therapy. We aimed to characterize acute and long-term functional and oncologic outcomes for severe ICI-myopathy-myocarditis, with focus on those treated with tocilizumab after failure of first-line therapy.
methodsWe performed a retrospective cohort study of patients admitted with severe (Common Terminology Criteria for Adverse Events [CTCAE] grade 3 or 4) ICI-myopathy-myocarditis managed at a Canadian tertiary referral center. Clinical characteristics, investigations, treatment, and functional and long-term cancer outcomes were summarized. Patients were stratified by treatment with tocilizumab after first-line therapy. Primary outcomes were in-hospital mortality and acute-phase survival. Secondary outcomes included functional status at last follow-up, final CTCAE grade, and oncologic response.
resultsEleven patients were identified. Eight patients received second-line tocilizumab at a median of 13 days after admission; weakness improved in all, but only 4 survived hospitalization. Among acute-phase survivors, median follow-up was 8.1 months, with final median CTCAE grade of 1. Limb strength normalized before oculobulbar weakness. Most survivors demonstrated partial oncologic response, followed by later disease progression at a median 8 months. No patients were rechallenged with ICI therapy. DISCUSSION: In severe ICI-myopathy-myocarditis refractory to first-line therapy, escalation with IL-6 receptor blockade was temporally associated with limb muscle strength improvement and favorable long-term functional outcomes among survivors. These findings support further evaluation of tocilizumab as second-line therapy in this high-risk population.
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