Hypertrophic Cardiomyopathy Considerations for the Managed Care Pharmacist

摘要

Hypertrophic cardiomyopathy (HCM) is often seen in patients as an autosomal dominant genetic heart disease with a variable clinical course. It is characterized by left ventricular hypertrophy, and with some patients, there is no evidence of a genetic etiology or presence of HCM in family members. Young age at diagnosis and the presence of a pathogenic or likely pathogenic sarcomere variant predict greater lifelong risk for stroke, heart failure, ventricular arrhythmia, atrial fibrillation, or mortality. Most individuals affected with HCM live to an average lifespan due to improvements in earlier diagnosis, sudden cardiac death risk stratification, family screening, pharmacologic therapy, devices, and invasive septal reduction therapy. Although these interventions have improved mortality, they are associated with significant costs and morbidities. There are burdensome costs related to genetic testing, family screening, implantable cardioverter-defibrillators, alcohol septal ablation, septal myectomy, pacemaker placement, and cardiac transplantation. In addition to these economic considerations, patients with HCM may experience a diminished health-related quality of life. Shared decision making between the patient and physician, use of multidisciplinary teams at HCM centers, and judicious use of exercise when appropriate have been shown to improve patient outcomes. Currently, treatments for HCM do not treat the underlying illness. Although not yet approved by the FDA, cardiac myosin inhibitors have recently shown promise in clinical trials to treat the underlying pathology of HCM. If approved by the FDA, managed care pharmacists should be ready to assess their safety and efficacy to improve the clinical burden and quality of life of those affected by HCM and reduce medical costs for these patients against standard of care. Long-term safety and efficacy data showing reductions in hospitalizations, morbidity, and mortality will be needed to determine their actual utility in managing HCM and ultimate place in therapy.