Outcomes in Patients With Immune Checkpoint Inhibitor-Related Myopathy and Prolonged Follow-Up

  • 0Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, MN.

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Summary

This summary is machine-generated.

Most patients with immune checkpoint inhibitor-related myopathy achieve good functional outcomes, though residual symptoms are common. Improvement continues over 12 months, but male sex and specific symptoms indicate a worse prognosis.

Area Of Science

  • Neurology
  • Immunology
  • Oncology

Background

  • Immune checkpoint inhibitors (ICIs) are crucial cancer therapies.
  • Immune-related adverse events (irAEs) are common, with myopathy being a frequent neurologic irAE.
  • Long-term outcomes of ICI-related myopathy are not well-established.

Purpose Of The Study

  • To evaluate the long-term outcomes of patients diagnosed with immune checkpoint inhibitor-related myopathy.
  • To identify factors associated with disease course and prognosis in ICI-related myopathy.

Main Methods

  • Retrospective review of patients with ICI-related myopathy at Mayo Clinic (2013-2024).
  • Comparison between patients with at least 6 months of follow-up and those who died within 2 months of onset.
  • Analysis of clinical presentation, treatment, and outcomes.

Main Results

  • Eighty-seven percent of patients achieved a favorable outcome (irAE grade ≤2) at last follow-up, with residual symptoms in 48%.
  • Most patients showed gradual improvement, even after treatment cessation.
  • Male sex, bulbar, and axial involvement were associated with a poorer prognosis.

Conclusions

  • Long-term functional outcomes for ICI-related myopathy are generally favorable, but residual symptoms persist in about half of patients.
  • Symptom improvement can continue for over 12 months.
  • Identifying specific clinical features can predict a more severe disease course, suggesting atypical pathology in refractory cases.

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