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

• Published in: Revista española de cardiología (English ed.) 2017-06, Vol.70 (6), p.444-450
• Main Authors: Saltijeral, Adriana, Pérez de Isla, Leopoldo, Alonso, Rodrigo, Muñiz, Ovidio, Díaz-Díaz, José Luis, Fuentes, Francisco, Mata, Nelva, de Andrés, Raimundo, Díaz-Soto, Gonzalo, Pastor, José, Pinilla, José Miguel, Zambón, Daniel, Pinto, Xavier, Badimón, Lina, Mata, Pedro
• Format: Article
• Language: English
• Subjects: Adolescent; Anticholesteremic Agents - therapeutic use; Child; Cholesterol, LDL - blood; Cholesterol, LDL - drug effects; Female; Follow-Up Studies; Guideline Adherence; Humans; Hyperlipoproteinemia Type II - blood; Hyperlipoproteinemia Type II - drug therapy; Hyperlipoproteinemia Type II - epidemiology; Incidence; Male; Prospective Studies; Registries; Spain - epidemiology; Treatment Outcome
• ISSN: 1885-5857; 1885-5857
• DOI: 10.1016/j.rec.2016.10.010

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. 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. 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 < 130mg/dL. Statin use was the only predictor of LDL-C goal attainment. 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.