Effect of Rosuvastatin on Carotid Intima-Media Thickness in Children with Heterozygous Familial Hypercholesterolemia: The CHARON Study

Background —Heterozygous familial hypercholesterolemia (HeFH) is an autosomal dominant disorder leading to premature atherosclerosis. Children with HeFH exhibit early signs of atherosclerosis, manifested by increased carotid intima-media thickness (IMT). In this study, we assessed the effect of 2-year treatment with rosuvastatin on carotid IMT in HeFH children. Methods —Children with HeFH (aged 6 to 4.9 mmol/L or >4.1 mmol/L in combination with other risk factors, received rosuvastatin for 2 years, starting at 5 mg once daily, with up-titration to 10 mg (aged 6 to Results —At baseline, mean (±standard deviation) carotid IMT was significantly greater for the 197 HeFH children compared with the 65 unaffected siblings (0.397±0.049 mm and 0.377±0.045 mm, respectively; p=0.001). During 2 years of follow-up, the change in carotid IMT was 0.0054 mm/y (95% CI: 0.0030 to 0.0082) in HeFH children and 0.0143 mm/y (95% CI: 0.0095 to 0.0192) in unaffected siblings (p =0.002). The end-of-study difference in mean carotid IMT between HeFH children and unaffected siblings after 2 years was no longer significant (0.408±0.043 mm and 0.402±0.042 mm, respectively; p =0.2). Conclusions —In HeFH children 6 years or older, carotid IMT was significantly greater at baseline compared with unaffected siblings. Rosuvastatin treatment for 2 years resulted in significantly less progression of increased carotid IMT in HeFH children than untreated unaffected siblings. As a result, no difference in carotid IMT could be detected between the two groups after 2 years of rosuvastatin. These findings support the value of early initiation of statin treatment for LDL-C reduction in children with HeFH. Clinical Trial Registration —URL: http://www.clinicaltrials.gov Unique identifier: NCT01078675.