Efficacy and safety of fluvastatin in children and adolescents with heterozygous familial hypercholesterolaemia.
OBJECTIVE
To assess whether early initiation of statin therapy for heterozygous familial hypercholesterolaemia favourably affects lipid profiles or vascular morphological changes.


METHODS
Children and adolescents aged 10-16 y with heterozygous familial hypercholesterolaemia were administered fluvastatin (80 mg/d) for 2 y in a single-arm two-centre study. Carotid B-mode intima-media thickness (IMT) and M-mode arterial wall stiffness (beta) were recorded. Eighty of the 85 enrolled subjects completed the trial.


RESULTS
The median decrease in low-density lipoprotein (LDL) cholesterol from baseline at last study visit was 33.9%; median decreases in total cholesterol, triglycerides and apolipoprotein B were 27.1%, 5.3% and 24.2%, respectively; the median increase in high-density lipoprotein (HDL) cholesterol was 5.3%. Changes in carotid arterial wall thickness and stiffness versus baseline were fractional and statistically non-significant (delta IMT -0.005 mm, 95% CI -0.018 to +0.007 mm, n=83; and delta beta = 0.017, 95% CI -0.219 to +0.253, n=79). Adverse events, all non-serious, were reported by 58 subjects (68.2%); four were suspected to be drug-related. Change in hormone levels and sexual maturation were appropriate for this age group.


CONCLUSIONS
Fluvastatin lowered LDL cholesterol, total cholesterol and apolipoprotein B levels effectively over a prolonged period in children and adolescents with heterozygous familial hypercholesterolaemia. Carotid IMT and wall stiffness remained largely unchanged.
van der Graaf
Anouk
A
Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands.
Nierman
Melchior C
MC
Firth
Jean C
JC
Wolmarans
Karen H
KH
Marais
A David
AD
de Groot
Eric
E
D000293:Adolescent; D050197:Atherosclerosis / Q000000981:diagnostic imaging / Q000517:prevention & control*; D002339:Carotid Arteries / Q000000981:diagnostic imaging*; D002648:Child; D003692:Delayed-Action Preparations; D005229:Fatty Acids, Monounsaturated / Q000627:therapeutic use*; D005260:Female; D000077340:Fluvastatin; D006801:Humans; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors / Q000627:therapeutic use*; D006938:Hyperlipoproteinemia Type II / Q000097:blood / Q000188:drug therapy*; D007211:Indoles / Q000627:therapeutic use*; D008055:Lipids / Q000097:blood; D008297:Male; D016037:Single-Blind Method; D016896:Treatment Outcome; D014463:Ultrasonography
D003692:Delayed-Action Preparations; D005229:Fatty Acids, Monounsaturated; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors; D007211:Indoles; D008055:Lipids; D000077340:Fluvastatin