Aims
Most heterozygous familial hypercholesterolemia (FH) patients require intensive lipid-lowering therapy (LLT) including PCSK9 inhibitors (PCSK9i) to reach current 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 of PCSK9i in FH patients in clinical practice setting.
Methods and Results
Spanish Familial Hypercholesterolemia 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 (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 mmol/L], 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.
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