Ezetimibe Treatment of Pediatric Patients with Hypercholesterolemia

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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).


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.


In this small retrospective series of children and adolescents with hypercholesterolemia, ezetimibe was safe and effective in lowering LDL-C levels.

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