Impact of Statin Treatment on the Clinical Fate of Heterozygous Familial Hypercholesterolemia

Aim: Familial hypercholesterolemia (FH) patients are at particular risk for premature coronary artery disease (CAD) caused by high levels of low density lipoprotein (LDL). Administration of statins enabled us to reduce LDL-C levels in heterozygous FH patients. To evaluate the impact of statins on the clinical fate of heterozygous FH, a retrospective study was performed. Methods: We analyzed the clinical influence of statins on age at the first clinical onset of CAD in 329 consecutive FH patients referred to the lipid clinic of the National Cardiovascular Center. Among 329 heterozygous FH patients, the onset of CAD was identified in 101. Results: The age at onset of CAD was 58.8 +/- 12.5 years in the 25 patients on statins at onset, significantly higher than that in the 76 patients not on statins (47.6 +/- 10.5 years) (p < 0.001). The average age at CAD onset was significantly higher after widespread use of statins (54.2 +/- 13.2 years in 48 patients; Group 1) compared to before October 1989 when statins were approved in Japan (46.9 +/- 9.6 years in 53 patients; Group 2, p=0.002). A significant difference was seen between Groups 1 and 2 in the variables, including sex, prevalence of smoking habit, LDL-C, and the use of statins, aspirin and probucol. After adjusting for these variables, only statin use was independently associated with the difference in age at CAD onset by multivariable analysis. Conclusion: Statins have improved the clinical course of patients with heterozygous FH.