Effect of Rosuvastatin on Carotid Intima-Media Thickness in Children with Heterozygous Familial Hypercholesterolemia: The CHARON Study
2017
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.
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