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Cited by 26Lipoproteins and Cardiovascular HealthLipid metabolism and disordersMedication Adherence and Compliance

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

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Alonso, Rodrigo, Arroyo-Olivares, Raquel, Muñiz-Grijalvo, Ovidio, Díaz-Díaz, Jose Luis, Muñoz-Torrero, Juan Sanchez, Romero, Manuel J, de Andrés, Raimundo, Zambón, Daniel +13 more·2023·European Journal of Preventive Cardiology
DOI 10.1093/eurjpc/zwac277
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Abstract

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.

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  • Studies of PCSK9 inhibitor monoclonal antibodies have reported first-year discontinuation rates approaching 50%.

    “Studies of PCSK9 inhibitor moAbs have reported first-year discontinuation rates approaching 50%, which can lead to suboptimal LDL cholesterol reduction compromising prevention of cardiovascular events.5, 6, 7, 8, 9, 10, 11, 12, 13”

    From Adherence to PCSK9 Inhibitors in Clinical Practice (2026) · cites this paper as [5]

    The Introduction (page unavailable) says some studies found that almost 40% discontinued PCSK9 inhibitors within six months. The Persistence with PCSK9 inhibitors section (page unavailable) reports 30–40% first-year non-persistence in non-FH populations, not a rate approaching 50%; SAFEHEART’s own first-year rate was 1.3%. (LLM, conf=0.86)

    Experimental — accuracy not yet measured

  • First-year discontinuation of PCSK9 inhibitor monoclonal antibodies can lead to suboptimal LDL cholesterol reduction compromising prevention of cardiovascular events.

    “Studies of PCSK9 inhibitor moAbs have reported first-year discontinuation rates approaching 50%, which can lead to suboptimal LDL cholesterol reduction compromising prevention of cardiovascular events.5, 6, 7, 8, 9, 10, 11, 12, 13”

    From Adherence to PCSK9 Inhibitors in Clinical Practice (2026) · cites this paper as [5]

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Authors
Alonso, R., Arroyo-Olivares, R., Muñiz-Grijalvo, O., Díaz-Díaz, J. L., Muñoz-Torrero, J. S., Romero, M. J., de Andrés, R., Zambón, D., Mañas, M. D., Fuentes-Jiménez, F., Aguado, R., Alvarez-Baños, P., Arrieta, F., Gonzalez-Bustos, P., Cepeda, J., Martin-Ordiales, M., Mosquera, D., Michan, A., de Isla, L. P., Argueso, R., & Mata, P.
Affiliations
  • Fundación Hipercolesterolemia Familiar , C/General Álvarez de Castro 14, 28010 Madrid , Spain
  • Hospital Universitario Virgen del Rocío (ES)
  • Department of Internal Medicine, Hospital Abente y Lago , A Coruña , Spain
  • Department of Internal Medicine, Hospital San Pedro de Alcántara , Cáceres , Spain
  • Department of Internal Medicine, Hospital Infanta Elena , Huelva , Spain
  • Department of Internal Medicine, Fundación Jiménez Díaz , Madrid , Spain
  • Hospital Clínic de Barcelona (ES)
  • Hospital General Universitario de Ciudad Real (ES)
  • Maimonides Biomedical Research Institute of Cordoba (IMIBIC), University of Cordoba, Reina Sofia University Hospital, CIBERObn , Córdoba , Spain
  • Hospital Universitario de León (ES)
  • Department of Endocrinology, Hospital Universitario de Burgos , Burgos , Spain
  • Hospital Universitario Ramón y Cajal (ES)
  • Department of Internal Medicine, Hospital Universitario Virgen de las Nieves , Granada , Spain
  • Hospital Vega Baja (ES)
  • Department of Internal Medicine, Hospital Universitario , Salamanca , Spain
  • Hospital San Pedro (ES)
  • Department of Internal Medicine, Hospital de Jerez , Jerez , Spain
  • Department of Cardiology, Hospital Clínico San Carlos, IDISSC , Madrid , Spain
  • Department of Endocrinology, Hospital Universitario Lucus Augusti , Lugo , Spain
Corresponding author
Mata, Pedro
ORCID
Venue
European Journal of Preventive Cardiology
Publisher
Oxford University Press (OUP)
Published
2022-11-23
Volume
30
Issue
4
Pages
320-328
ISSN
  • 2047-4873 (ISSN-L)
  • 2047-4881
DOI
10.1093/eurjpc/zwac277
OpenAlex
W4309821617
Cited by
26
References
1
Citations by year
2026: 3 · 2025: 6 · 2024: 7 · 2023: 9 · 2022: 1
FWCI
5.52
Type
journal article
Language
en
Open access
Licence
Locations
  • European Journal of Preventive Cardiology (publishedVersion, cc-by-nc)
  • PubMed (publishedVersion)
  • Universidad de Córdoba Insitutional Repository (Universidad de Córdoba) (submittedVersion, cc-by)
Funders
  • Fundación Hipercolesterolemia (G03/181, FIS PI12/01289)
  • Instituto de Salud Carlos III (08-2008)
  • Centro Nacional de Investigaciones Cardiovasculares
Topics
  • Health Sciences › Medicine › Surgery › Lipoproteins and Cardiovascular Health
  • Health Sciences › Medicine › Cardiology and Cardiovascular Medicine › Lipid metabolism and disorders
  • Health Sciences › Medicine › Family Practice › Medication Adherence and Compliance
Keywords
PCSK9 inhibitors, familial hypercholesterolemia, treatment persistence, LDL cholesterol lowering, quality of life, lipid-lowering therapy, long-term treatment effectiveness, atherosclerotic cardiovascular disease prevention, heterozygous familial hypercholesterolemia, real-world clinical practice, treatment discontinuation
MeSH terms
Proprotein Convertase 9, PCSK9 Inhibitors, Anticholesteremic Agents, Female, Humans, Hyperlipoproteinemia Type II, Cholesterol, LDL, Male, Prospective Studies, Quality of Life, Cohort Studies, Hydroxymethylglutaryl-CoA Reductase Inhibitors

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