Ten years of statin therapy started in childhood: efficacy, safety, compliance and tolerability in patients with familial hypercholesterolemia (AfterTen-study)

In the LIPIDS trial, children aged 10-18 with Familial hypercholesterolemia
(FH), an hereditary disorder characterized by elevated levels of low-density
lipoprotein cholesterol (LDL-C) causing premature atherosclerotic
cardiovascular events, have been treated with pravastatin 20-40 mg. This agent
was safe and effective in lowering LDL-C and intima media thickness (IMT)
progression, a surrogate marker for atherosclerosis, during the study. However
the effect of such early treatment initiation, mainly with respect to
atherosclerosis progression in the long term, is not exactly known.

This study will evaluate the effect of approximately 10 years of pravastatin
treatment on IMT in young adults with FH. Furthermore, safety parameters and
psychological aspects with respect to early treatment initiation will be
investigated.

Patients who participated in the LIPIDS trial (n=204) as well as their
non-affected siblings (n*80), will be requested to visit our center once.
During the visit we will obtain medical history, perform a physical
examination, measure IMT and draw blood. Furthermore, a questionnaire on
medication use and psychological aspects will be filled out. FH patients will
be asked for permission to contact their physicians who treat them with respect
to FH in order to verify data on medication use and medical history.

Patients will pay only one visit of approximately 3 hours for blood sampling
(50 ml), medical history and physical examination, filling out a questionnaire
with respect to psychological aspects and IMT measurement. We consider this of
minimal burden for the participants. Patients who are not treated according to
current best practice will benefit from participation since their treatment
regimens will be optimized according to current guidelines.