Persistence with long-term PCSK9 inhibitor treatment and its effectiveness in familial hypercholesterolaemia: data from the SAFEHEART study.

Autor:	Alonso R; Fundación Hipercolesterolemia Familiar, C/General Álvarez de Castro 14, 28010 Madrid, Spain.; Center for Advanced Metabolic Medicine and Nutrition, Santiago de Chile, Fundación hipercolesterolemia Familiar, Madrid, Spain., Arroyo-Olivares R; Fundación Hipercolesterolemia Familiar, C/General Álvarez de Castro 14, 28010 Madrid, Spain., Muñiz-Grijalvo O; Department of Internal Medicine, Hospital Virgen del Rocío, Sevilla, Spain., Díaz-Díaz JL; Department of Internal Medicine, Hospital Abente y Lago, A Coruña, Spain., Muñoz-Torrero JS; Department of Internal Medicine, Hospital San Pedro de Alcántara, Cáceres, Spain., Romero MJ; Department of Internal Medicine, Hospital Infanta Elena, Huelva, Spain., de Andrés R; Department of Internal Medicine, Fundación Jiménez Díaz, Madrid, Spain., Zambón D; Lipid Unit, Hospital Clinic, Barcelona, Spain., Mañas MD; Department of Internal Medicine, Hospital Universitario, Ciudad Real, Spain., Fuentes-Jiménez F; Maimonides Biomedical Research Institute of Cordoba (IMIBIC), University of Cordoba, Reina Sofia University Hospital, CIBERObn, Córdoba, Spain., Aguado R; Department of Endocrinology, Hospital General de León, León, Spain., Alvarez-Baños P; Department of Endocrinology, Hospital Universitario de Burgos, Burgos, Spain., Arrieta F; Department of Endocrinology, Hospital Ramón y Cajal, Madrid, Spain., Gonzalez-Bustos P; Department of Internal Medicine, Hospital Universitario Virgen de las Nieves, Granada, Spain., Cepeda J; Department of Internal Medicine, Hospital Comarcal Vega Baja, Orihuela, Spain., Martin-Ordiales M; Department of Internal Medicine, Hospital Universitario, Salamanca, Spain., Mosquera D; Department of Internal Medicine, Hospital San Pedro, Logroño, Spain., Michan A; Department of Internal Medicine, Hospital de Jerez, Jerez, Spain., de Isla LP; Department of Cardiology, Hospital Clínico San Carlos, IDISSC, Madrid, Spain., Argueso R; Department of Endocrinology, Hospital Universitario Lucus Augusti, Lugo, Spain., Mata P; Fundación Hipercolesterolemia Familiar, C/General Álvarez de Castro 14, 28010 Madrid, Spain.
Jazyk:	angličtina
Zdroj:	European journal of preventive cardiology [Eur J Prev Cardiol] 2023 Mar 01; Vol. 30 (4), pp. 320-328.
DOI:	10.1093/eurjpc/zwac277
Abstrakt:	Aims: Most heterozygous familial hypercholesterolaemia (FH) patients require intensive lipid-lowering therapy (LLT) including PCSK9 inhibitors (PCSK9is) to reach current low-density lipoprotein cholesterol (LDL-C) goals. Persistence with chronic treatment is important to reduce the burden of atherosclerotic cardiovascular disease. We analysed persistence, efficacy, and impact on quality of life (QoL) of PCSK9i in FH patients in clinical practice setting. Methods and Results: Spanish Familial Hypercholesterolaemia Cohort Study (SAFEHEART) is an open, prospective study in genetically defined FH patients in Spain. Patients ≥18 years of age (n = 696, 46% females) on stable LLT treated with PCSK9i were analysed. Median LDL-C at starting PCSK9i was 145 mg/dL [interquartile range (IQR), 123-177], 3.8 mmol/L (IQR 3.2-4.6). After a median follow up of 3.7 years (IQR 2.3-4.8), 27 patients (4%) discontinued PCSK9i treatment: 5 temporarily (0.7%) and 22 permanently (3.2%). Persistence with PCSK9i was 96.1% in the whole period. Median LDL-C levels and % LDL-C reduction attained after 1 year of treatment and in the last follow-up visit were 63 mg/dL (IQR 43-88), 1.6 mmol/L (IQR 1.1-2.23); 61 mg/dL (IQR 44-82), 1.6 mmol/L (IQR 1.1-2.1); 57.6% (IQR 39.5-69); and 58% (IQR 44-68), respectively. 2016 and 2019 ESC/EAS LDL-C goals were attained by 77 and 48% of patients, respectively, at the last follow-up visit (P < 0.001). Mean QoL score increased slightly in the first year and remained stable. Conclusion: Long-term persistence with PCSK9i in FH patients is very high, with a good QoL. Effectiveness in LDL-C reduction and LDL-C goal achievement dramatically improved with PCSK9i in this high-risk population in clinical practice setting. Trial Registration: ClinicalTrials.gov number NCT02693548. Competing Interests: Conflict of interest: R.A. received honorary fees for talks and advisory boards from Amgen, Sanofi, MSD, Tecnofarma, Abbott, Saval. O.M.G. received honoraria for speaker or researcher activities from Merck Sharp and Dohme, Amgen, and Sanofi. J.L.D.D. received honoraria for speaker or researcher activities from Merck Sharp and Dohme, Amgen, and Sanofi. L.P.I. received honoraria for speaker or researcher activities from Merck Sharp and Dohme, Astra Zeneca, Esteve, Amgen, and Sanofi. P.M. received honoraria for advisory boards and received research grants from Amgen and Sanofi. (© The Author(s) 2022. Published by Oxford University Press on behalf of the European Society of Cardiology.)
Databáze:	MEDLINE
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