Atorvastatin and cardiac hypertrophy and function in hypertrophic cardiomyopathy: a pilot study.

Background: Hypertrophic cardiomyopathy (HCM) is a genetic paradigm of cardiac hypertrophy. Cardiac hypertrophy is a major determinant of risk of sudden death and morbidity in HCM. Treatment with statins reverses hypertrophy in animal models of HCM. Thus, statins may afford therapeutic benefits in HCM.

Methods: We performed a feasibility study with atorvastatin to gather the pre-requisite data for designing randomized efficacy studies.

Results: We screened 32 patients with HCM in 18months. Twenty-one patients met the study criteria and consented to participate. The demographics and echocardiographic phenotype of those who did and those who did not participate were not significantly different. We treated the participants with escalating doses of atorvastatin (20, 40 and 80mgday(-1) ) for 2years. We performed ECG and echocardiography and measured plasma lipids, liver enzymes, creatine kinase and B-type natriuretic peptide levels before and after 3, 6, 12 and 24months of therapy. Fifteen, 12 and 11 patients completed 6, 12 and 24months of therapy respectively. Six patients discontinued atorvastatin because of perceived lack of benefit. We stopped atorvastatin in 4 patients because of modest elevations in liver enzymes, creatine kinase or back pain. The characteristics of those who did or did not complete the study were not significantly different. The mean plasma low-density lipoprotein-cholesterol level was reduced by 55%. However, echocardiographic indices of cardiac hypertrophy and function remained unchanged.

Conclusions: The findings illustrated the challenges that will be encountered in designing efficacy studies to test the potential beneficial effects of atorvastatin in human HCM.