TREATMENT OF HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA WITH PROBUCOL

Abstract

Ten patients with homozygous familial hypercholesterolemia were treated with probucol for 15-21 mo. Xanthomas regressed or disappeared in most patients, angina lessened in 2/3 and the ECG improved in half of those with pretreatment abnormalities. The mean fall in serum total cholesterol levels after 18 months of therapy was 27%. High-density lipoprotein cholesterol levels fell transiently in the early mo. of therapy but then returned to the pretreatment range. Plasma probucol levels varied from 20-90 .mu.g/ml, indicating good compliance, but did not correlate with the clinical or hypocholesterolemic responses. Probucol was well tolerated by both child and adult patients. The effects of probucol in this therapeutically resistant disorder were better than expected. Considered in relation to efficacy, safety, tolerance and convenience probucol appears to be the most satisfactory treatment for homozygous familial hypercholesterolemia currently available.