Design and baseline data of a pediatric study with rosuvastatin in familial hypercholesterolemia

作者：Kusters D Meeike^*; Hutten Barbarba A; McCrindle Brian W; Cassiman David; Francis Gordon A; Gagne Claude; Gaudet Daniel; Morrison Katherine M; Langslet Gisle; Kastelein John J; Wiegman Albert

来源：Journal of Clinical Lipidology, 2013, 7(5): 408-413.

DOI：10.1016/j.jacl.2013.06.010

• Pediatrics
• Hypercholesterolemia
• Statins
• Carotid intima-media thickness

BACKGROUND: Statin therapy is recommended for children with familial hypercholesterolemia (FH), but most children do not reach treatment targets. %26lt;br%26gt;OBJECTIVE: Here we present the design and results at baseline of the ongoing CHARON study, to evaluate the safety and efficacy of rosuvastatin. %26lt;br%26gt;METHODS: This study comprises an international 2-year open label, titration-to-goal study in 198 children with heterozygous PH aged 6 to 18 years, with rosuvastatin in a maximum dose of 10 mg (%26lt;10 years of age) or 20 mg (older children). In addition, 64 unaffected siblings were enrolled as controls. The primary efficacy outcome is the change from baseline in low-density lipoprotein cholesterol, and the secondary outcome is the change in carotid intima-media thickness (c-IMT) in patients with FH compared with their siblings. The primary safety outcomes are growth and sexual maturation; secondary outcomes are the change in other lipoprotein levels and the incidence of adverse events, discontinuation rates, and abnormal laboratory values. %26lt;br%26gt;RESULTS: At baseline, mean age of patients with FH was 12.1.+/- 3.3 years, 44% were boys, and mean low-density lipoprotein cholesterol levels were 6.1 +/- 1.3 mmol/L (235.9 +/- 48.7 mg/dL). Mean c-IMT was 0.399 mm (95% CI, 0.392-0.406 mm) in children with FE versus 0.377 (95% CI, 0.366-0.388 mm) in unaffected siblings (P = .001). %26lt;br%26gt;CONCLUSIONS: At baseline, as expected according to on previous observations, children with FE proved to have a greater c-IMT than their healthy siblings. These differences had already occurred at a very young age, which emphasizes the importance of considering early statin initiation in this high-risk population.

• 出版日期2013-10