Treatment of homozygous familial hypercholesterolaemia with probucol.

Ten patients with homozygous familial hypercholesterolaemia were treated with the recently introduced drug probucol (Lurselle; Mer-National) for 15-21 months. Xanthomas regressed or disappeared in most patients, angina lessened in two-thirds of cases 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 months of therapy but then returned to the pretreatment range. Plasma probucol levels varied between 20 micrograms/ml and 90 micrograms/ml, indicating good compliance, but did not correlate with the clinical or hypocholesterolaemic responses. Probucol was well tolerated by 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 hypercholesterolaemia currently available.