1. Efficacy of PCSK9 inhibitors in the treatment of heterozygous familial hypercholesterolemia: A clinical practice experience
- Author
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Patricia Rubio, Raimundo de Andrés, Raquel Arroyo-Olivares, Pedro Mata, María Dolores Mañas, Francisco Fuentes-Jiménez, Marta Casañas, Pilar Alvarez-Baños, Francisco Arrieta, Ovidio Muñiz-Grijalvo, Rodrigo Alonso, Leopoldo Pérez-Isla, José María Cepeda, Marta Dieguez, Juan Francisco Sánchez Muñoz-Torrero, José Luis Díaz-Díaz, Daniel Zambón, Pablo Gonzalez-Bustos, Rocío Aguado, and Rosa Argüeso
- Subjects
Male ,medicine.medical_specialty ,Injections, Subcutaneous ,Endocrinology, Diabetes and Metabolism ,Disease ,Familial hypercholesterolemia ,030204 cardiovascular system & hematology ,Antibodies, Monoclonal, Humanized ,Cohort Studies ,Hyperlipoproteinemia Type II ,03 medical and health sciences ,0302 clinical medicine ,Internal medicine ,Internal Medicine ,medicine ,Humans ,Prospective Studies ,030212 general & internal medicine ,PCSK9 Inhibitors ,Prospective cohort study ,Aged ,Alirocumab ,Nutrition and Dietetics ,business.industry ,Cholesterol, LDL ,Middle Aged ,medicine.disease ,Clinical Practice ,Evolocumab ,Treatment Outcome ,Cohort ,Female ,lipids (amino acids, peptides, and proteins) ,Cardiology and Cardiovascular Medicine ,business ,Follow-Up Studies - Abstract
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.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.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 2020.433 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) p0.001, 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.PCSK9 inhibitors on top of maximum lipid-lowering treatment significantly reduced LDL-C levels in patients with familial hypercholesterolemia and improved the achievement of LDL-C targets.
- Published
- 2021