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Consecución de objetivos terapéuticos de colesterol LDL en niños y adolescentes con hipercolesterolemia familiar. Registro longitudinal SAFEHEART

Publicado en:REVISTA ESPANOLA DE CARDIOLOGIA. 70 (6): 444-450 - 2017-06-01 70(6), DOI: 10.1016/j.recesp.2016.10.012

Autores: Saltijeral Cerezo, Adriana; Pérez de Isla, Leopoldo; Alonso, Rodrigo; Muñiz Grijalvo, Ovidio; Díaz Díaz, José Luis; Mata Pariente, Nelva; Andrés Cañas, Raimundo; Díaz Soto, Gonzalo; Pastor Rosado, José; Pinilla Alemán, Jose Miguel; Zambón, Daniel; Pintó Sala, Xavier; Badimon Maestro, Lina; Mata López, Pedro; Fuentes Jiménez, Francisco

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© 2016 Sociedad Española de Cardiología Introduction and objectives Little is known about the characteristics of persons with familial hypercholesterolemia (FH) younger than 18 years, the lipid-lowering therapy used in these patients, and the lipid goals reached in real life. Our aim was to evaluate the achievement of low-density lipoprotein cholesterol (LDL-C) treatment goals in FH patients younger than 18 years enrolled in a large national registry. Methods We analyzed patients younger than 18 years enrolled in a large ongoing registry of molecularly-defined patients with FH in Spain. The attainment of guideline-recommended plasma LDL-C goals at entry and follow-up was analyzed in relation to the use of lipid-lowering therapy. Results We enrolled 392 individuals younger than 18 years. Of these, 217 were molecularly-diagnosed FH patients and had a complete follow-up. The median follow-up time was 4.69 years (interquartile range, 2.48-6.38 years), 68.2% of FH patients were on statins, and 41.5% patients had LDL-C < 130 mg/dL. Statin use was the only predictor of LDL-C goal attainment. Conclusions This study shows that a high proportion of FH patients younger than 18 years have high LDL-C levels and fail to achieve recommended LDL-C targets. Statin use was the only independent predictor of LDL-C goal achievement. No safety concerns were detected during follow-up. These results indicate that many FH patients are not adequately controlled and that there is still room for treatment improvement. Full English text available from: www.revespcardiol.org/en

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