Journal
JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY
Volume 67, Issue 11, Pages 1278-1285Publisher
ELSEVIER SCIENCE INC
DOI: 10.1016/j.jacc.2016.01.008
Keywords
cardiovascular disease; LDL-receptor mutations; lipid-lowering therapy; low-density lipoprotein cholesterol
Categories
Funding
- Fundacion Hipercolesterolemia Familiar
- Instituto de Salud Carlos III (ISCIII) [G03/181, FIS PI12/01289]
- Centro Nacional de Investigacion Cardiovascular (CNIC) [08-2008]
- Merck Sharp Dohme
- AstraZeneca
- AMGEN
- AEGERION
- ISIS
- Sanofi
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BACKGROUND Familial hypercholesterolemia (FH) is the most common genetic disorder associated with premature atherosclerotic cardiovascular disease (ASCVD). There are sparse data on attainment of treatment targets; large registries that reflect real-life clinical practice can uniquely provide this information. OBJECTIVES We sought to evaluate the achievement of low-density lipoprotein cholesterol (LDL-C) treatment goals in FH patients enrolled in a large national registry. METHODS The SAFEHEART study (Spanish Familial Hypercholesterolemia Cohort Study) is a large, ongoing registry of molecularly defined patients with heterozygous FH treated in Spain. The attainment of guideline-recommended plasma LDL-C goals at entry and follow-up was investigated in relation to use of lipid-lowering therapy (LLT). RESULTS The study recruited 4,132 individuals (3,745 of whom were >= 18 years of age); 2,752 of those enrolled were molecularly diagnosed FH cases. Mean follow-up was 5.1 +/- 3.1 years; 71.8% of FH cases were on maximal LLT, and an LDL-C treatment target <100 mg/dl was reached by only 11.2% of patients. At follow-up, there was a significant increase in the use of ezetimibe, drug combinations with statins, and maximal LLT. The presence of type 2 diabetes mellitus, a defective allele mutation, ezetimibe use, and the absence of previous ASCVD were predictors of the attainment of LDL-C goals. CONCLUSIONS Despite the use of intensified LLT, many FH patients continue to experience high plasma LDL-C levels and, consequently, do not achieve recommended treatment targets. Type of LDL-receptor mutation, use of ezetimibe, coexistent diabetes, and ASCVD status can bear significantly on the likelihood of attaining LDL-C treatment goals. (C) 2016 by the American College of Cardiology Foundation.
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