Background Evolocumab and alirocumab, protein convertase subtilisin/kexin type 9 (PCSK9) inhibitors, are indicated for the treatment of familial hypercholesterolaemia and atherosclerotic cardiovascular disease.

Purpose The objectives of the study were to evaluate the profile of medication use, effectiveness and safety of the treatment.

Material and methods Retrospective observational study of patients treated with evolocumab or alirocumab from September 2016 to the present.

The collected variables were: sex, age, statins tolerance, lipid profile, lipid-lowering therapies coadyuvant, duration and reason for treatment. Effectiveness was evaluated as low-density lipoprotein-cholesterol (LDL-C) reduction. The safety profile has been determined according to the adverse reactions.

Results Twenty patients were included, 12 male; follow-up (median, range): 60 (19–109) weeks; age: 55 (33–74) years. Two patients were excluded because follow-up was less than 4 weeks. The therapeutic indications were: familial hypercholesterolaemia 61% (n=11) and atherosclerotic cardiovascular disease 39% (n=7). All of them had been previously treated with statins until resistance (maximum dose) or intolerance was developed. The treatment received was: evolocumab (72%) and alirocumab (28%). The average of basal LDL-C and post-treatment was 164 mg/dL (108–369) and 78 mg/dL (39–153), respectively. Patients treated with evolocumab decreased LDL-C levels by 67% and patients treated with alirocumab decreased LDL-C levels by 29%. Fifty-five per cent of the patients received PCSK9 inhibitor treatment combined with statin and ezetimibe. Currently, all patients continue with the treatment.

Conclusion Clinical criteria for treatment initiation should be considered individually. The results of the study evidence the effectiveness of both treatments, being superior in the group treated with evolocumab. The treatment’s safety profile is very favourable. Studies with a larger sample size are required to obtain representative data and determine the optimal duration of the treatment.

References and/or acknowledgements No conflict of interest.