Reduction of Serum Cholesterol in Heterozygous Patients with Familial Hypercholesterolemia

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Abstract
We studied the effects of the bile acid sequestrant cholestyramine, alone and in combination with the experimental agent compactin (ML-236B), a competitive inhibitor of 3-hydroxy-3-methylglutaryl coenzyme A reductase, on serum levels of lipoproteins in 10 heterozygous patients with familial hypercholesterolemia. After cholestyramine treatment alone for 2 to 16 months, serum total and low-density lipoprotein cholesterol decreased by 20 and 28 per cent, respectively. With the addition of compactin for 12 weeks there was a 39 per cent total decrease in serum cholesterol from the control value — from 356±14 to 217±10 mg per deciliter (9.27±0.36 to 5.64±0.26 mmol per liter [mean ±S.E.M.]; P<0.001) — and a 53 per cent decrease in low-density lipoprotein cholesterol — from 263±13 to 125±10 mg per deciliter (6.84±0.34 to 3.25±0.26 mmol per liter; P<0.001). High-density lipoprotein cholesterol, which had increased during cholestyramine treatment, remained at its higher level. No adverse effects were observed. If long-term safety can be demonstrated, the compactin–cholestyramine regimen may prove useful in heterozygous familial hypercholesterolemia. (N Engl J Med. 1983; 308:609–13.)