Ezetimibe Treatment of Pediatric Patients with Hypercholesterolemia

Objective To review the efficacy of ezetimibe monotherapy for treatment of hypercholesterolemia in pediatric patients. Study design This is a retrospective review of all pediatric patients who received ezetimibe monotherapy as treatment for hypercholesterolemia and for whom follow-up clinical and lipid results were available. Of 36 identified patients, 26 had lipoprotein profiles suggestive of familial hypercholesterolemia (FH), and 10 had profiles suggestive of familial combined hyperlipidemia (FCHL). Results After a mean 105 days of treatment with ezetimibe (range, 32-175 days), total cholesterol (TC) levels decreased from 7.3 ± 1.0 mmol/L to 5.7 ± 1.0 mmol/L ( P < .0001), and low-density lipoprotein cholesterol (LDL-C) levels decreased from 5.3 ± 0.9 mmol/L to 3.9 ± 0.8 ( P < .0001) in patients with FH. In patients with FCHL, TC levels decreased from 6.4 ± 2.0 mmol/L to 5.6 ± 0.4 mmol/L ( P ≤ .002), and LDL-C levels decreased from 4.7 ± 1.0 mmol/L to 3.8 ± 0.6 mmol/L ( P ≤ .005). For all patients, the mean decrease in individual LDL-C values was 1.5 ± 0.9 mmol/L or 28%. There was no significant change in triglyceride or high-density lipoprotein cholesterol levels with ezetimibe. Patients were maintained on ezetimibe with no adverse effects attributable to the medication for as long as 3.5 years. At a mean of 13.6 months (range, 1-44 months) after the initiation of ezetimibe, LDL-C levels remained decreased at 4.0 ± 0.6 mmol/L. Conclusions In this small retrospective series of children and adolescents with hypercholesterolemia, ezetimibe was safe and effective in lowering LDL-C levels.