Design and baseline data of a pediatric study with rosuvastatin in familial hypercholesterolemia.
BACKGROUND
Statin therapy is recommended for children with familial hypercholesterolemia (FH), but most children do not reach treatment targets.


OBJECTIVE
Here we present the design and results at baseline of the ongoing CHARON study, to evaluate the safety and efficacy of rosuvastatin.


METHODS
This study comprises an international 2-year open label, titration-to-goal study in 198 children with heterozygous FH aged 6 to 18 years, with rosuvastatin in a maximum dose of 10 mg (<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.


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 FH versus 0.377 (95% CI, 0.366-0.388 mm) in unaffected siblings (P = .001).


CONCLUSIONS
At baseline, as expected according to on previous observations, children with FH 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.
Department of Vascular Medicine, Academic Medical Center, Amsterdam, The Netherlands; Department of Pediatrics, Academic Medical Center, Amsterdam, The Netherlands. Electronic address: d.m.kusters@amc.uva.nl.
D Meeike
DM
Kusters
Barbarba A
BA
Hutten
Brian W
BW
McCrindle
David
D
Cassiman
Gordon A
GA
Francis
Claude
C
Gagné
Daniel
D
Gaudet
Katherine M
KM
Morrison
Gisle
G
Langslet
John J
JJ
Kastelein
Albert
A
Wiegman
D000293:Adolescent; D002648:Child; D005260:Female; D005464:Fluorobenzenes / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000188:drug therapy*; D008297:Male; D011743:Pyrimidines / Q000009:adverse effects / Q000627:therapeutic use*; D000068718:Rosuvastatin Calcium; D012449:Safety; D035781:Siblings; D013449:Sulfonamides / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
D005464:Fluorobenzenes; D011743:Pyrimidines; D013449:Sulfonamides; D000068718:Rosuvastatin Calcium
Carotid intima-media thickness; Hypercholesterolemia; Pediatrics; Statins