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Attainment of LDL-Cholesterol Treatment Goals in Patients With Familial Hypercholesterolemia

2016; Elsevier BV; Volume: 67; Issue: 11 Linguagem: Inglês

10.1016/j.jacc.2016.01.008

1558-3597

Leopoldo Pérez de Isla, Rodrigo Alonso, Gerald F. Watts, Nelva Mata, Adriana Saltijeral Cerezo, Ovidio Muñiz-Grijalvo, Francisco Fuentes, José Luis Díaz-Díaz, Raimundo de Andrés, Daniel Zambón, Patricia Rubio, Miguel Ángel Barba-Romero, Pedro Luis Ruíz Sáenz, Juan Francisco Sánchez Muñoz-Torrero, Ceferino Martínez‐Faedo, José Pablo Miramontes‐González, Lina Badimón, Pedro Mata, Rocío Aguado, Fátima Almagro, Francisco Arrieta, Miguel Ángel Elvira Barba, Ángel Brea, José María Cepeda, Raimundo de Andrés, Gonzalo Dí­az, José Luis Díaz-Díaz, Francisco Fuentes, Jesús Galiana, Juan Antonio Garrido, Luis Irigoyen, Laura Manjón, Alberto Martín, Mar Piedecausa, Ceferino Martínez‐Faedo, Marta Mauri, Pablo Miramontes, Ovidio Muñiz-Grijalvo, Francisca Pereyra, Leire Pérez, Xavier Pintó, Pedro Pujante, Enrique Ruíz, Pedro Luis Ruíz Sáenz, Juan F. Plaza, Jose I. Vidal, Rosa Argüeso, Daniel Zambón,

Lipoproteins and Cardiovascular Health

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. We sought to evaluate the achievement of low-density lipoprotein cholesterol (LDL-C) treatment goals in FH patients enrolled in a large national registry. 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). 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. 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.