Double-blind, placebo-controlled, cross-over trial of probucol in heterozygous familial hypercholesterolaemia.

Ten patients with heterozygous familial hypercholesterolaemia (FH) were given probucol 500 mg b.d. or placebo for 3 months in a randomised order in a double-blind cross-over trial. There was a 14% decrease in serum cholesterol concentration due to a reduction in both low density lipoprotein (LDL) cholesterol and high density lipoprotein (HDL) cholesterol. The subfraction of HDL most affected was HDL2. Reductions in serum LDL cholesterol concentration exceeding 20% were obtained in 3 (30%) of the patients. The magnitude of the change in LDL cholesterol concentration was related to the level of serum HDL2 cholesterol without therapy and to the magnitude of its decrease on probucol. Intravenous intralipid tolerance was unaffected by probucol administration. Serum apolipoprotein B concentration decreased less with probucol than did that of serum LDL cholesterol.