4CPS-017 Evaluation of effectiveness and safety of alirocumab and evolocumab in real-world settings

<h3>Background and Importance</h3> Proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors are monoclonal antibodies that bind to PCSK9, regulating low-density lipoprotein (LDL) metabolism and LDL receptor degradation. <h3>Aim and Objectives</h3> The aim of this study was to evaluate effectiveness and safety of PCSK9 inhibitors in management of primary hypercholesterolaemia and mixed dyslipidemia. <h3>Material and Methods</h3> Observational, descriptive, and retrospective study including patients treated with PCSK9 inhibitors from 2021 to 2023. Analysed sociodemographic variables were sex and age. Clinical variables included diagnosis, treatment, concomitant high-dose statin therapy (yes/no), and baseline and final LDL levels. Effectiveness was assessed by LDL reduction, treatment persistence, cardiovascular-related hospitalisations, and overall survival. Safety was evaluated by the occurrence of adverse reactions (ARs). High-dose statin therapy was defined as atorvastatin 80 mg/day or rosuvastatin 20 mg/day or higher. <h3>Results</h3> 33 patients were included with a median age of 57 (21–80) years and 50% were male. Primary hypercholesterolaemia was diagnosed in 53.12%, while 46.88% had mixed dyslipidemia. Alirocumab was given to 59.38% of patients, and 40.63% received Evolocumab. Half of the patients received high-dose statin therapy. The median baseline LDL level was 132 mg/dl (range 62–320), and the median final LDL level was 67 mg/dl (range 25–158). LDL levels were reduced by at least 50% in 39.39% of patients. Median persistence, hospitalisation, and overall survival were not reached. ARs occurred in 15.15% of patients, with upper respiratory tract infections (40%), arthralgia (20%), hypersensitivity (20%), and pruritus (20%). <h3>Conclusion and Relevance</h3> PCSK9 inhibitors proved effective and safe for managing primary hypercholesterolaemia and mixed dyslipidemia. However, due to the small sample size, the results are mostly limited to surrogate markers like LDL reduction. This reduction was less than in clinical trials, where a 50% decrease was noted at 24 months; in our study, only 40% of patients achieved this. The difference may stem from lower adherence to lifestyle and medication outside clinical trials. Safety data showed good tolerability, with fewer reported adverse reactions, possibly due to underreporting in real-world settings. Larger studies with longer follow-up are needed to fully understand these drugs’ impact. <h3>References and/or Acknowledgements</h3> <h3>Conflict of Interest</h3> No conflict of interest