Efficacy of PCSK9 inhibitors in the treatment of heterozygous familial hypercholesterolemia: A clinical practice experience


Por: Alonso, R, Muniz-Grijalvo, O, Diaz-Diaz, J, Zambon, D, de Andres, R, Arroyo-Olivares, R, Fuentes-Jimenez, F, Munoz-Torrero, J, Cepeda, J, Aguado, R, Alvarez-Banos, P, Casanas, M, Dieguez, M, Manas, M, Rubio, P, Argueso, R, Arrieta, F, Gonzalez-Bustos, P, Perez-Isla, L, Mata, P and SAFEHEART Investigators

Publicada: 1 nov 2021 Ahead of Print: 1 nov 2021
Resumen:
Background: PCSK9 inhibitors are a treatment option for patients with familial hypercholesterolemia not on low-density lipoprotein cholesterol goals despite the use of maximally tolerated high intensity-statins dose. Objective: To evaluate the efficacy of alirocumab and evolocumab in LDL-C reduction and targets attainment in patients with heterozygous familial hypercholesterolemia in clinical practice setting. Methods: SAFEHEART is an open, long-term prospective study of a cohort of subjects with molecular diagnosis of familial hypercholesterolemia. This study analyze subjects >= 20 years of age on stable lipid-lowering therapy, who received PCSK9 inhibitors during the period 2016 to January 2020. Results: 433 patients (mean age 55 years, 53% male, 39% with cardiovascular disease) were included and followed-up for a median of 2.5 years (IQR 1.6-3.0). Median LDL-C level prior to PCSK9 inhibitors was 145 mg/dL (IQR 125-173). The addition of PCSK9 inhibitors (211 alirocumab, 222 evolocumab) reduced LDL-C by 58% (IQR 41-70) p <0.001, in men and women, achieving a median LDL-C level of 62 mg/dL (IQR 44-87) without differences between both PCSK9 inhibitors. Out of them 67% with and 80% without cardiovascular disease reached 2016 ESC/EAS LDL-C targets, and 46% very high risk and 50% high risk patients achieved 2019 ESC/EAS LDL-C goals. Independent predictor factors for attainment of 2019 ESC/EAS LDL-C goals were to be male, smoking and the use of statins with ezetimibe. Both inhibitors were well tolerated. Conclusions: PCSK9 inhibitors on top of maximum lipid-lowering treatment significantly reduced LDLC levels in patients with familial hypercholesterolemia and improved the achievement of LDL-C targets. (C) 2021 National Lipid Association. Published by Elsevier Inc. All rights reserved.

Filiaciones:
Alonso, R:
 Ctr Adv Metab Med & Nutr, Av Las Condes 9460,501, Santiago, Chile

Mata, P:
 Fdn Hipercolesterolemia Familiar, Gen Alvarez de Castro 14, E-2800 Madrid, Spain

Hosp Virgen Rocio, Internal Med Dept, Seville, Spain.
Hosp Abente & Lago, Internal Med Dept, La Coruna, Spain.
Hosp Clin Barcelona, Lipid Unit, Barcelona, Spain.
Fdn Jimenez Diaz, Internal Med, Madrid, Spain.
Hosp Univ Reina Sofia, Lipid & Atherosclerosis Unit, IMBic, CIBERObn, Cordoba, Spain.
Hosp San Pedro Alcantara, Internal Med Dept, Caceres, Spain.
Hosp Comarcal Vega Baja, Internal Med Dept, Orihuela, Spain.
Hosp Gen Leon, Dept Endocrinol, Leon, Spain.
Hosp Univ Burgos, Dept Endocrinol, Burgos, Spain.
Hosp San Pedro, Internal Med Dept, Logrono, Spain.
Hosp Cabuenes, Dept Endocrinol, Gijon, Spain.
Hosp Univ, Internal Med Dept, Ciudad Real, Spain.
Hosp Jerez de la Frontera, Internal Med Dept, Cadiz, Spain.
Hosp Univ Lucus Augusti, Dept Endocrinol, Lugo, Spain.
Hosp Ramon & Cajal, Dept Endocrinol, Madrid, Spain.
Hosp Univ Virgen Nieves, Internal Med Dept, Granada, Spain.
Hosp Clin San Carlos, Dept Cardiol, IDISSC, Madrid, Spain.
ISSN: 19332874





JOURNAL OF CLINICAL LIPIDOLOGY
Editorial
Elsevier BV, STE 800, 230 PARK AVE, NEW YORK, NY 10169 USA, Países Bajos
Tipo de documento: Article
Volumen: 15 Número: 4
Páginas: 584-592
WOS Id: 000729988800010
ID de PubMed: 34052174

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