Ezetimibe as Monotherapy in the Treatment of Hypercholesterolemia in Children and Adolescents

Background: A prospective study was conducted to evaluate low-density lipoprotein-cholesterol (LDL-C) lowering efficacy and tolerability of ezetimibe as monotherapy in children and adolescents with polygenic hypercholesterolemia (PH) or familial hypercholesterolemia (FH). Methods and Results: Children with PH (n = 6) or FH (n = 11) aged 5-15 years were consecutively enrolled to receive ezetimibe as monotherapy at 10 mg/day for 11.3 ± 7.3 and 15.9 ± 10.1 months, respectively. Plasma biochemical and lipid profiles were assessed before and after treatment. Ezetimibe significantly lowered total cholesterol (TC) and LDL-C in patients with PH and FH: TC from 260.5 ± 12.4 to 180.0 ± 21.6 mg/dl (p = 0.02) and from 315.3 ± 41.8 to 233.3 ± 36.8 mg/dl (p = 0.003), respectively, and LDL-C from 177.1 ± 17.7 to 102.6 ± 16.7 mg/dl (p = 0.02) and from 243.0 ± 41.8 to 170.0 ± 29.8 mg/dl (p = 0.003), respectively. However, high-density lipoprotein-cholesterol (HDL-C) only decreased significantly (from 58.1 ± 10.0 to 49.3 ± 9.1 mg/dl) (p <0.01) in patients with FH and remained unaltered in patients with PH. Triglyceride levels remained unchanged in both groups. Biochemical profile (hemogram, transaminases, creatinine, calcium, phosphorus and liposoluble vitamins A and E) remained unchanged; no adverse effects were observed. Conclusions: Our data show that ezetimibe as monotherapy significantly lowered TC and LDL-C in children with PH and FH.