We report the case of a 76-year-old man with germline CTLA-4 deficiency manifesting as multivessel coronary artery disease, heart failure with mid-range ejection fraction, and an aortic root aneurysm. New York Heart Association (NYHA) class 3 functional limitation was disproportionate to ischemic burden despite guideline-directed medical therapy. Statin intolerance prompted initiation of evolocumab, a PCSK9 inhibitor. Within 3 weeks, the patient's symptoms improved to NYHA class 1, suggesting a rapid immunomodulatory effect. This case illustrates targetable cardiomyopathy mediated by CTLA-4 deficiency, supports exploring PCSK9 inhibition for the management of immune-driven heart failure, and emphasizes genetic evaluation in unexplained cardiomyopathy.
Bannister et al. (Thu,) studied this question.