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Persistence with long-term PCSK9 inhibitor treatment and its effectiveness in familial hypercholesterolaemia: data
Rodrigo Alonso1,2, Raquel Arroyo-Olivares1, Ovidio Muñiz-Grijalvo3
1Fundación Hipercolesterolemia Familiar, C/General Álvarez de Castro 14, 28010 Madrid, Spain.
Insights
PCSK9 inhibitors show high persistence and effectiveness in lowering LDL-C for familial hypercholesterolaemia patients. This treatment improves quality of life and significantly reduces cardiovascular disease risk in clinical practice.
Area of Science:
- Cardiology
- Genetics
- Pharmacology
Background:
- Familial hypercholesterolaemia (FH) necessitates intensive lipid-lowering therapy (LLT).
- PCSK9 inhibitors (PCSK9i) are crucial for achieving low-density lipoprotein cholesterol (LDL-C) goals in FH.
- Treatment persistence is vital for mitigating atherosclerotic cardiovascular disease.
Purpose of the Study:
- To analyze the persistence, efficacy, and quality of life (QoL) impact of PCSK9i in FH patients.
- To evaluate PCSK9i effectiveness in a real-world clinical setting.
Main Methods:
- Prospective analysis of 696 genetically defined FH patients on stable LLT treated with PCSK9i.
- Median follow-up of 3.7 years.
- Assessment of LDL-C levels, persistence rates, and QoL scores.
Main Results:
- High persistence rate of 96.1% with PCSK9i over the study period.
- Significant LDL-C reduction, with median levels reaching 61 mg/dL at last follow-up.
- Achievement of 2016/2019 ESC/EAS LDL-C goals by 77% and 48% of patients, respectively.
- Slight improvement and stabilization of QoL scores.
Conclusions:
- Long-term persistence with PCSK9i in FH patients is very high.
- PCSK9i demonstrates significant effectiveness in LDL-C reduction and goal attainment in high-risk FH populations.
- PCSK9i treatment positively impacts QoL in FH patients.
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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