Statins for Children with Familial Hypercholesterolemia

*Dinara Sadykova, Liliia Galimova, Evgeniia Slastnikova, Zulfiia Khabibrakhmanova and Natalya Guseva*

#### **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.

**Keywords:** children, familial hypercholesterolemia, total cholesterol, low density lipoprotein efficacy, treatment, statins, side effects

#### **1. Introduction**

Hypercholesterolemia occupies an important place among the factors of cardiovascular mortality [1]. It is known that the level of lipids in 40–60% of cases is due to genetic reasons [2]. Familial hypercholesterolemia is one of the most common hereditary diseases: its prevalence is 1: 200–1: 500 in the general population [3]. The estimated number of people with a heterozygous form of the disease in Russia should be about 1 million [4]. Despite the high urgency of early detection of the disease, in our country it is diagnosed in less than 1% of the expected number of patients. Diagnosis and initiation of therapy for the disease in childhood are considered optimal before complications such as aortic stenosis, atherosclerotic changes in the arterial walls appear. Statins are recommended for treatment as first-line drugs, but experience with their use in children is limited and requires special analysis.

Statins are 3-hydroxy-3-methyl coenzyme A reductase inhibitors that limit the rate of endogenous cholesterol synthesis. This leads to a decrease in the content of intracellular cholesterol and the level of circulating LDL-C in the blood. In addition to the lipid-lowering effect, statins affect atherosclerotic plaque (reduce its size, stabilize the surface, thereby reducing the risk of rupture and ulceration), as well as inflammatory factors and endothelial function (pleiotropic effects) [5]. The main goal of prescribing statins in familial hypercholesterolemia is to reduce the risk and rate of development of atherosclerosis and coronary heart disease in order to delay the onset of cardiovascular accidents as much as possible. It should be noted that the use of statins among the adult population, as a rule, is not in doubt, while the pharmacotherapy of hypercholesterolemia in pediatrics raises questions from doctors and parents regarding its effectiveness, long-term prospects and possible complications.
