Background and Importance Evolocumab, an inhibitor of proprotein convertase subtilin-kexin type 9, represents an alternative therapeutic option for individuals who exhibit intolerance to standard low-density lipoprotein cholesterol (LDL-C) treatments or fail to attain desired LDL-C levels.

Aim and Objectives This study aims to assess the effectiveness, safety and adherence to evolocumab among patients with hypercholesterolemia.

Material and Methods Observational, retrospective, and multidisciplinary study that included patients who started treatment with evolocumab in a tertiary hospital between July 2016 and August 2022. Data variables (clinical history and dispensing program) were sex, age, indication, statins treatment, evolucumab dosage, treatment duration, LDL-C levels at baseline, 3, 6, 12 and 36 months, adverse effects (AEs) and adherence (medication possession rate). SPSS-27 statistical program (Wilcoxon test) was used to compare the decrease in LCL-C levels at different times.

Results The study enrolled 63 patients (52.4% women), with an average age at initiation of 61.8 (SD:11.1) years. The primary diagnoses included familial hypercholesterolemia (57.1%), established cardiovascular disease (33.3%) or both (9.5%). 63.5% of patients were intolerant to statins, 1.6% had contraindications, and 34.9% received statins at maximum tolerated doses without achieving target LDL-C levels. Dosage was 140 mg/14 days, with an average treatment duration of 3.0 (SD:1.6) years and an adherence rate of 91.3 (SD:14.9)%. The average LDL-C levels was 169.9 (SD:57.5)mg/dl, 84.9 (SD: 62.6) mg/dl, 77.2 (SD: 47.5)mg/dl, 75.7 (SD: 39.0) mg/dl and 84.0 (SD: 44.5) mg/dl at basal, 3, 6, 12 and 36 months, respectively. These LDL-C levels were significantly reduced (p<0.01) when compared to basal. Currently the majority (85.7%) of patients continue their treatment, 1.6% lost to follow-up, and 12.7% discontinued due to death (4.8%), AEs (6.3%) and lack of response (1.6%). Only four patients had AEs (headache; pseudo catarrhal symptoms, haematomas, spasms; anaphylaxis; skin reaction, diarrhoea and myopathies), and evolocumab was withdrawn in all of them.

Conclusion and Relevance Evolocumab emerges as a compelling therapeutic option for LDL-C reduction and cardiovascular risk mitigation, particularly for patients with statin intolerance or inadequate statin response. The results obtained in our real clinical practice (55.4% decrease in LDL-C levels at 12 months) were similar to those of the pivotal clinical trials. Further research is warranted to ascertain its impact on major cardiovascular events in real-world settings.

Conflict of Interest No conflict of interest.