Statins for Children with Familial Hypercholesterolemia

Abstract

Familial hypercholesterolemia (FH) is the most common genetic disorder in the world. It is characterized by increased level of total cholesterol (TC), low-density lipoproteins (LDL-C) since childhood. The diagnosis and initiation of therapy are optimal in childhood before complications (aortic stenosis, atherosclerotic changes in the arterial walls) appear. The initiation of lipid-lowering therapy in FH since childhood is important to reduce the cumulative effect of LDL-C, to increase patient’s life expectancy. Statins are recommended as first-line drugs for treatment with monitoring of the recommended clinical, biochemical markers under the supervision of a physician. However, due to limited experience, there are differing opinions among clinicians regarding the age of initiation of lipid-lowering therapy. This review is an attempt to critically study the available data from the world literature concerning the use of statins in children with FH, their effectiveness, safety. It is important to determine the endpoints for determining the effectiveness of statins, such as lowering LDL-C, assessing the thickness of the intima-media complex. The frequency of occurrence of possible side effects in children is considered - diabetes mellitus, hepatotoxicity, muscle pain and others. There is a need to continue randomized trials to prove the lifelong benefit of low LDL-C in patients with FH.