20-Year follow-up of statins in children with familial hypercholesterolemia

Autor: Michel H. Hof, D Meeike Kusters, Albert Wiegman, John J.P. Kastelein, Jaap W. Groothoff, Ilse K. Luirink, Barbara A. Hutten, Eric de Groot
Přispěvatelé: AGEM - Amsterdam Gastroenterology Endocrinology Metabolism, Paediatric Nephrology, AGEM - Inborn errors of metabolism, APH - Quality of Care, APH - Methodology, ACS - Diabetes & metabolism, ACS - Atherosclerosis & ischemic syndromes, Graduate School, Paediatric Metabolic Diseases, Epidemiology and Data Science, ARD - Amsterdam Reproduction and Development, Vascular Medicine, APH - Health Behaviors & Chronic Diseases, APH - Aging & Later Life, ACS - Heart failure & arrhythmias, ACS - Pulmonary hypertension & thrombosis
Jazyk: angličtina
Rok vydání: 2019
Předmět:
Zdroj: New England journal of medicine, 381(16), 1547-1556. Massachussetts Medical Society
ISSN: 0028-4793
Popis: BACKGROUND Familial hypercholesterolemia is characterized by severely elevated low-density lipoprotein (LDL) cholesterol levels and premature cardiovascular disease. The short-term efficacy of statin therapy in children is well established, but longer follow-up studies evaluating changes in the risk of cardiovascular disease are scarce. METHODS We report a 20-year follow-up study of statin therapy in children. A total of 214 patients with familial hypercholesterolemia (genetically confirmed in 98% of the patients), who were previously participants in a placebo-controlled trial evaluating the 2-year efficacy and safety of pravastatin, were invited for follow-up, together with their 95 unaffected siblings. Participants completed a questionnaire, provided blood samples, and underwent measurements of carotid intima-media thickness. The incidence of cardiovascular disease among the patients with familial hypercholesterolemia was compared with that among their 156 affected parents. RESULTS Of the original cohort, 184 of 214 patients with familial hypercholesterolemia (86%) and 77 of 95 siblings (81%) were seen in follow-up; among the 214 patients, data on cardiovascular events and on death from cardiovascular causes were available for 203 (95%) and 214 (100%), respectively. The mean LDL cholesterol level in the patients had decreased from 237.3 to 160.7 mg per deciliter (from 6.13 to 4.16 mmol per liter)-a decrease of 32% from the baseline level; treatment goals (LDL cholesterol
Databáze: OpenAIRE