Studies of statin treatment in patients (≤20 years) with heterozygous hypercholesterolaemia, that reported clinical data as primary outcomes, were eligible for inclusion in the review. Case reports were excluded from the review.
Study designs included in the review were randomised controlled trials (RCTs), prospective case series, a parallel matched non-randomised trial, a cross-over trial and a time series comparison. Interventions included in the review were: pravastatin, simvastatin, lovastatin, atorvastatin; further details are reported in the review.
For the RCTs, follow-up ranged from six to 96 weeks (median duration of treatment was 27 weeks). All RCTs identified participants either by a minimum low density lipoprotein (4.0mmol/L) and at least one parent having a clinical, or molecular diagnosis of familial hypercholesterolaemia, or a positive low density lipoprotein receptor gene mutation.
In included studies, participants' ages ranged from four to 18 years (eight to 18 years for the RCTs). Most studies included both genders but two studies included only males. Primary outcomes measures were mean change from baseline in low density lipoprotein, high density lipoprotein, and total cholesterol and triglycerides. A number of other outcomes, including laboratory biochemistry, cardiovascular and adverse events, were also reported.
One reviewer reviewed titles and abstracts and selected articles for inclusion in a preliminary short-list, and two reviewers independently selected studies for inclusion in the review from this short-list. Any disagreements were resolved through consensus between three reviewers.