Background Monoclonal antibodies (mAb) arilocumab and evolocumab are protein convertase subtilisin/kexin type 9 inhibitors (PCSK9) for the primary treatment of hypercholesterolaemia or mixed dyslipidaemia:

• in combination with other lipid lowering therapies unable to reach low density lipoprotein (LDL-c) goals (<100 mg/dL);

• alone or in combination with other lipid lowering therapies in patients statin intolerant or statin contraindicated.

Purpose To evaluate the effectiveness, safety and cost of alirocumab and evolocumab.

Material and methods This was a retrospective observational study from April to September 2016. Data collected: sex, age, diagnosis, previous/concomitant treatment and duration of treatment. The study evaluated: (1) effectiveness: total cholesterol (total-c) and LDL-c (electronic clinical review: MambrinoXXI); (2) safety: established adverse events (AE) reported for patients in the pharmacy outpatient unit; (3) cost: cost/patient/year.

Results 12 patients were included (92% men), median age 58 years (range 25–78). Diagnosis: 41% dyslipidaemia, 25% hypercholesterolaemia, 17% hyperlipidaemia and 17% heart disease. 50% patients received arilocumab and 50% evolocumab. All patients had been treated with statins before mAb therapy. In 42% of cases statins had to be removed, mainly because of myositis (80%). The remainder of the patients were not statin-intolerant but LDL-c goals were not achieved. During mAb therapy, 17% of patients were treated with evolocumab as monotherapy; 50% with arilocumab or evolocumab plus two lipid lowering therapies (statin and fenofibrate or ezetimibe); 17% with mAb plus fenofibrate and ezetimibe; 8% with mAb and statin; and 8% with mAb and fenofibrate. At the end of the study, median duration of treatment was 15 weeks (range 11–19) and all patients continued mAb treatment. Effectiveness: before treatment with mAb, mean total-c and LDL-c values were 208.60 mg/dL and 140.7 mg/dL, respectively. At the end of the study, these values were 125.3 mg/dL and 68.9 mg/dL, with a mean reduction of 39% and 35%, respectively. No AE were reported. Estimated cost in our hospital (first year): €5000/patient.

Conclusion New lipid lowering drugs seem to be a new therapeutic alternative for hypercholesterolaemia or mixed dyslipidaemia when statins and/or other lipid lowering therapies are not effective or contraindicated. However, effectiveness is only valuable with LDL-c data, regardless of cardiovascular morbidity and mortality effects. Hence it is necessary to conduct long term studies to check any other effects of these drugs beyond reduction of LDL-c values.

No conflict of interest