Attainment of LDL Cholesterol Treatment Goals in Children and Adolescents With Familial Hypercholesterolemia. The SAFEHEART Follow-up Registry

作者：Saltijeral Adriana; Perez de Isla Leopoldo; Alonso Rodrigo; Muniz Ovidio; Luis Diaz Diaz Jose; Fuentes Francisco; Mata Nelva; de Andres Raimundo; Diaz Soto Gonzalo; Pastor Jose; Miguel Pinilla Jose; Zambon Daniel; Pinto Xavier; Badimon Lina; Mata Pedro

来源：Revista Espanola de Cardiologia, 2017, 70(6): 444-450.

DOI：10.1016/j.rec.2016.10.010

• Familial hypercholesterolemia
• Low-density lipoprotein cholesterol goals
• Lipid-lowering therapy
• Cardiovascular disease
• Children
• Adolescents
• Statins

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.

• 出版日期2017-6