ArticleNeurology(R) neuroimmunology & neuroinflammation2025
Outcomes in Patients With Immune Checkpoint Inhibitor-Related Myopathy and Prolonged Follow-Up.
Article in Neurology(R) neuroimmunology & neuroinflammation, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
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Who cites it
5 citing papers in PubMed.
- The Immune Checkpoint Inhibitors Journey: From Early Promise to Lasting Impact.Journal of immunotherapy and precision oncology · 2026Review
- Myocardial Immune Niches in Homeostasis and Inflammation.Immunological reviews · 2026Review
- Tocilizumab for severe immune checkpoint inhibitor myopathy-myocarditis: acute and long-term outcomes.Journal of neurology · 2026Article
- Immune checkpoint inhibitor-related myotoxicity in lung cancer: a decade-long real-world pharmacovigilance analysis of the FAERS database.Frontiers in pharmacology · 2026Article
- Immune Checkpoint Inhibitor-Induced Central and Peripheral Neurotoxicity: Case Series and Literature Review.Cureus · 2025Article
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Authors and funding
11 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND AND
objectivesMyopathy is one of the most common neurologic immune-related adverse events (irAEs) reported after treatment with immune checkpoint inhibitor (ICI) cancer immunotherapies. Current knowledge on disease course relates to short-term outcomes, and long-term outcome data are lacking. The aim of this study was to evaluate the long-term outcomes of patients with ICI-related myopathy.
methodsWe reviewed Mayo Clinic patients diagnosed with ICI-related myopathy between 2013 and 2024 with at least 6 months of follow-up; we compared them with patients who died because of myopathy within 2 months from disease onset.
resultsTwenty-three patients were identified; the median follow-up duration was 13 months (range 6-76). The median age at presentation was 73 years (range 34-87), and 57% were male. The most common presentations were ocular involvement (78%, including oculomotor and ptosis), followed by proximal limb (74%), axial (61%), and bulbar (48%) weakness. Myocarditis was present in 35%. At disease nadir, 43% had an irAE grade >2. All patients were treated with corticosteroids; 61% received additional immunosuppressive/immunomodulatory treatment in the acute setting. The median treatment duration was 5 months (range 1-17). At the last follow-up, 87% of patients had a favorable outcome (irAE grade ≤2), yet residual symptoms/signs were common (48%). Gradual improvement was observed in most patients, even after immunosuppressive/immunomodulatory treatment was discontinued. The only patient with a truly refractory course had biopsy findings of nemaline rods. Compared with patients with long-term follow-up, patients who died within 2 months (N = 9) were more commonly men (57% vs 100%, DISCUSSION: Approximately half of the patients with ICI-related myopathy and long-term follow-up had some residual symptoms/signs, despite good functional outcomes. Symptoms continue to improve over 12 months regardless of immunosuppressive/immunomodulatory treatment duration. Male sex and axial and bulbar symptoms/signs were all associated with a terminal disease course. A prolonged refractory disease course might suggest an atypical pathology.
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