Endocrine Abstracts

Homozygous familial hypercholesterolemia (HoFH) represents a potentially fatal condition that is caused by low density lipoprotein (LDL) cholesterol receptor deficiency. Statins alone are not effective in HoFH. In recent years, semiselective or selective LDL cholesterol apheresis systems have been shown to prolong life. However, applying LDLC apheresis therapy, recent evidence also suggests that more intense LDL cholesterol reduction is associated with more clinical benefit. Ezetimibe is the first of a new class of cholesterol absorbtion inhibitors that potently inhibits dietary and biliary cholesterol absorbption.

The aim of this study was tested whether ezetimibe can induce a further reduction of LDL cholesterol levels in patients with high dose atorvastatin therapy or LDL cholesterol apheresis.

Four patients were recruited from our apheresis unit. All patients were treated by atorvastatin 80 mg/day and regular LDL cholesterol apheresis treatment every two weeks. LDL cholesterol apheresis was carried out using the cascade filtration (Dideco, Italy). Ezetimibe was given at a dose of 10 mg/day after a meal once every day for 6 weeks.

At the end of treatment period, changes in LDL cholesterol −10.6%, in total cholesterol −9.53% in VLDL cholesterol −9.83% were detected significantly (P<0.01) (P<0.05). Changes in HDL cholesterol and triglyceride were not significantly detected. No adverse events were observed with ezetimibe treatment.

Our results show that LDL cholesterol reduction by ezetimibe is a safe and effective therapy in patient with combined therapy with high dose atorvastatin and LDL cholesterol apheresis for HoFH.