Genetic migraine disorders and the response to calcitonin gene‐related peptide antagonist treatment

Calcitonin gene-related peptide (CGRP) is a potent cerebral vasodilator and part of the trigeminal migraine cascade. Newer migraine therapies target CGRP signaling for both acute and preventative management of headache. In this series, we present two cases of genetic conditions, of which migraine is a key feature, responsive to CGRP antagonist therapy. A 31-year-old female with mitochondrial encephalopathy with lactic acidosis and stroke-like episodes and a phenotype of chronic migraine with visual aura, and a 62-year-old female with cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy with a phenotype of chronic migraine and side-locked headache with cranial autonomic symptoms. Each experienced a significant reduction in the frequency of their migraine attacks with monoclonal antibody treatment against CGRP. In this case series, we add to the growing body of evidence that CGRP-blocking medications are safe and effective in some heritable neurological disorders in which vasomodulation is a common underlying pathology. To our knowledge, we present the first cases of galcanezumab use in an individual with mitochondrial encephalopathy with lactic acidosis and stroke-like episodes and eptinezumab in an individual with cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy.