title: Attainment of LDL Cholesterol Treatment Goals in Children and Adolescents With Familial Hypercholesterolemia. The SAFEHEART Follow-up Registry.
abstract: OBJECTIVE
  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 < 130mg/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.
authors.affiliation: Sección de Cardiología, Hospital Universitario del Tajo, Universidad Alfonso X el Sabio, Aranjuez, Madrid, Spain. Electronic address: adicerezo@gmail.com.
authors.forename: Adriana
authors.initials: A
authors.lastname: Saltijeral
authors.affiliation: Servicio de Cardiología, Hospital Clínico San Carlos, Universidad Complutense de Madrid, IDISSC, Madrid, Spain.
authors.forename: Leopoldo
authors.initials: L
authors.lastname: Pérez de Isla
authors.affiliation: Servicio de Medicina Interna, Clínica las Condes, Santiago de Chile, Chile.
authors.forename: Rodrigo
authors.initials: R
authors.lastname: Alonso
authors.affiliation: Servicio de Medicina Interna, Hospital Virgen del Rocío, Sevilla, Spain.
authors.forename: Ovidio
authors.initials: O
authors.lastname: Muñiz
authors.affiliation: Servicio de Medicina Interna, Hospital Abente y Lago, A Coruña, Spain.
authors.forename: José Luis
authors.initials: JL
authors.lastname: Díaz-Díaz
authors.affiliation: Unidad de Lípidos y Aterosclerosis, IMIBIC, Hospital Universitario Reina Sofía, Universidad de Córdoba, Córdoba, Spain.
authors.forename: Francisco
authors.initials: F
authors.lastname: Fuentes
authors.affiliation: Departamento de Epidemiología, Consejería de Sanidad, Comunidad de Madrid, Madrid, Spain.
authors.forename: Nelva
authors.initials: N
authors.lastname: Mata
authors.affiliation: Servicio de Medicina Interna, Fundación Jiménez Díaz, Madrid, Spain.
authors.forename: Raimundo
authors.initials: R
authors.lastname: de Andrés
authors.affiliation: Departamento de Endocrinología, Hospital Clínico, Valladolid, Spain.
authors.forename: Gonzalo
authors.initials: G
authors.lastname: Díaz-Soto
authors.affiliation: Servicio de Pediatría, Hospital General Universitario de Elche, Alicante, Spain.
authors.forename: José
authors.initials: J
authors.lastname: Pastor
authors.affiliation: Centro de Salud San Miguel de Salinas, San Miguel de Salinas, Alicante, Spain.
authors.forename: José Miguel
authors.initials: JM
authors.lastname: Pinilla
authors.affiliation: Clínica de Lípidos, Servicio de Endocrinología, Hospital Clínic, Barcelona, Spain.
authors.forename: Daniel
authors.initials: D
authors.lastname: Zambón
authors.affiliation: Servicio de Medicina Interna, Hospital Universitario de Bellvitge, L'Hospitalet de Llobregat, Barcelona, Spain.
authors.forename: Xavier
authors.initials: X
authors.lastname: Pinto
authors.affiliation: Centro de Investigación Cardiovascular, Instituto Catalán de Ciencias Cardiovasculares, IIB-Sant Pau, Barcelona, Spain.
authors.forename: Lina
authors.initials: L
authors.lastname: Badimón
authors.affiliation: Fundación Hipercolesterolemia Familiar, Madrid, Spain.
authors.forename: Pedro
authors.initials: P
authors.lastname: Mata
mesh_terms: D000293:Adolescent; D000924:Anticholesteremic Agents / Q000627:therapeutic use*; D002648:Child; D008078:Cholesterol, LDL / Q000097:blood* / Q000187:drug effects; D005260:Female; D005500:Follow-Up Studies; D019983:Guideline Adherence; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000097:blood / Q000188:drug therapy* / Q000453:epidemiology; D015994:Incidence; D008297:Male; D011446:Prospective Studies; D012042:Registries; D013030:Spain / Q000453:epidemiology; D016896:Treatment Outcome
chemical_list: D000924:Anticholesteremic Agents; D008078:Cholesterol, LDL
keywords: Adolescentes; Adolescents; Cardiovascular disease; Children; Enfermedad cardiovascular; Estatinas; Familial hypercholesterolemia; Hipercolesterolemia familiar; Lipid-lowering therapy; Low-density lipoprotein cholesterol goals; Niños; Objetivos de colesterol unido a lipoproteínas de baja densidad; Statins; Terapia hipolipemiante