Endocrine Abstracts

Introduction: PCSK9 inhibitors (iPCSK9) have previously been evaluated through controlled clinical trials showing up to 60% reduction in LDL cholesterol concentrations. However, we still do not have enough data regarding the real life experience of the treatment.

Objectives: 1) Describe patients with hypercholesterolemia treated with iPCSK9 in clinical practice.

2) Evaluate treatment efficacy and safety.

Methods: Retrospective observational study. Inclusion criteria: patients treated with iPCSK9 at the Alicante University General Hospital. Primary end point: changes in LDL cholesterol 3 and 6 months after the start of iPCSK9. Secondary end point: changes in total cholesterol (TC), HDL cholesterol and triglycerides (TG). Other variables: Occurrence of adverse events. Statistical analysis: descriptive (mean ± S.D., median [P25–P75]), Wilcoxon; P<0.05; SPSS v22.0.

Results: 24 patients (50% women, age 60±12 years, BMI 27±3 kg/m²). 62.5% heterozygous familial hypercholesterolemia, 58% onset cardiovascular disease, 21% both. At baseline 66% patients were taking combination ezetimibe-statins therapy (21% rosuvastatin 20 mg/d, 4% rosuvastatin 10 mg/d, 13% rosuvastatin 40 mg/d, 21% atorvastatin 80 mg/d), 21% statin therapy (8% rosuvastatin 20 mg/d, 8% atorvastatin 80 mg/d), 4% monotherapy with ezetimibe 10 mg/d. Evolocumab 140 mg was prescribed in 62.5% of cases and alirocumab 75 mg in 37.5%. Primary end point: 50% reduction in LDL [16–61%] at 3 months, 55% [42–74%] at 6 months (P<0.01). Secondary end point: 38% reduction in TC [21–44%] at 3 months, 41% [21–44%] at 6 months (P<0.01). Non-significant 7% decrease in TG at 3 months, 3% at 6 months. There were also no significant changes in HDL. 5 of the 24 patients (21%) presented mild adverse events: 1 recurrent respiratory tract infections, 2 flu-like syndrome, 1 hypertransaminasemia, 1 pruritus. None of them demonstrated increase in plasma glucosa levels, or showed neurocognitive symptoms.

Conclusion: In short-term real life studies, the addition of PCSK9 inhibitors to their previous treatment leads to improvement in metabolic control with an adequate safety profile.