Yahel Segal, Georgios Mangioris, Michel Toledano, Lisa A. Kottschade, Eoin P. Flanagan, Andrew McKeon, Ronen Stoff, Elie Naddaf, Sean J. Pittock, Divyanshu Dubey, Αναστασία Ζεκερίδου
BACKGROUND AND OBJECTIVES: Myopathy 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. METHODS: We 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. RESULTS: = 0.04, respectively). 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.