Results of intensive long-term treatment of familial hypercholesterolemia

Fifty-seven patients with familial hypercholesterolemia (FH) with mean age of 48 years (range 30 to 69), participated in a follow-up examination 5.5 years after the completion of a 1-year trial with lovastatin, cholestyramine, probucol, or omega-3 fatty acids. The goals were to record quality of life, compliance to treatment, adverse effects, and clinical outcome. The quality of life was similar to that in a Norwegian reference population, The factors causing most distress to patients were keeping a diet low in saturated fats, taking medication, and fear of death, The medication was mostly prescribed in maximum dosages. At follow-up, the reduction in total cholesterol was 36% (p <0.05), low-density lipoprotein (LDL) cholesterol 38% (p <0.05), triglycerides 20% (p <0.05) compared with being on diet therapy only, High-density lipoprotein (HDL) cholesterol increased 8% (p <0.05). Intake of saturated and monounsaturated fat increased 1.5% and 1.7% (p <0.05), respectively; polyunsaturated fat was unchanged, Three patients experienced myocardial infarction, of whom 2 died and 1 developed angina pectoris. Before the start of lovastatin treatment, 27 coronary events occured per 1,000 patient-years in this group compared with 12 events per 1,000 patient-years thereafter, OF 28 patients reporting adverse events, 4 discontinued lovastatin and 3 discontinued cholestyramine. Several practical and psychological difficulties were associated with FH. Long-term intensive lipid-lowering therapy was possible in FH outpatients without loss of effect and with good compliance to therapy, Intensive therapy, today is, however, not sufficient for many FH patients to reach a therapeutic goal of LDL cholesterol <4.0 mmol/L. More potent lipid-lowering agents are needed. (C) 1996 by Excerpta Medica, Inc.