title: Results of intensive long-term treatment of familial hypercholesterolemia.
abstract: 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 occurred 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.
authors.affiliation: Medical Department A, Rikshospitalet, Oslo, Norway.
authors.forename: K
authors.initials: K
authors.lastname: Retterstøl
authors.forename: M
authors.initials: M
authors.lastname: Stugaard
authors.forename: C
authors.initials: C
authors.lastname: Gørbitz
authors.forename: L
authors.initials: L
authors.lastname: Ose
mesh_terms: D000328:Adult; D000368:Aged; D000924:Anticholesteremic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D009320:Atrial Natriuretic Factor / Q000097:blood; D002784:Cholesterol / Q000097:blood; D002792:Cholestyramine Resin / Q000627:therapeutic use; D005260:Female; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000097:blood / Q000188:drug therapy*; D008148:Lovastatin / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D011341:Probucol / Q000627:therapeutic use; D011788:Quality of Life; D016896:Treatment Outcome
chemical_list: D000924:Anticholesteremic Agents; D002792:Cholestyramine Resin; D009320:Atrial Natriuretic Factor; D002784:Cholesterol; D008148:Lovastatin; D011341:Probucol