Tocilizumab for severe immune checkpoint inhibitor myopathy-myocarditis: acute and long-term outcomes.
G, C., B, M.R., M, A., B, P., & G, B. (2026). Tocilizumab for severe immune checkpoint inhibitor myopathy-myocarditis: acute and long-term outcomes.. Journal of neurology. https://doi.org/10.1007/s00415-026-13923-w
G C, B MR, M A, B P, G B. Tocilizumab for severe immune checkpoint inhibitor myopathy-myocarditis: acute and long-term outcomes.. Journal of neurology. 2026; doi: 10.1007/s00415-026-13923-w
G C, B MR, M A, et al. Tocilizumab for severe immune checkpoint inhibitor myopathy-myocarditis: acute and long-term outcomes.[J]. Journal of neurology. 2026. DOI: 10.1007/s00415-026-13923-w.
@article{g2026,
author = {Chaggar G and Mulvany-Robbins B and Anaka M and Putko B and Beecher G},
title = {Tocilizumab for severe immune checkpoint inhibitor myopathy-myocarditis: acute and long-term outcomes.},
journal = {Journal of neurology},
year = {2026},
doi = {10.1007/s00415-026-13923-w},
note = {PMID: 42240908},
}
TY - JOUR AU - Chaggar G AU - Mulvany-Robbins B AU - Anaka M AU - Putko B AU - Beecher G TI - Tocilizumab for severe immune checkpoint inhibitor myopathy-myocarditis: acute and long-term outcomes. T2 - Journal of neurology PY - 2026 DO - 10.1007/s00415-026-13923-w AN - PMID:42240908 ER -
BACKGROUND: Immune 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. METHODS: We 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. RESULTS: Eleven 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.