Background In our community, alirocumab and evolocumab, first-in-class proprotein convertase subtilisin–kexin type-9 inhibitors (PCSK9-I), have been authorised by the public health system for the treatment of patients with uncontrolled familial hypercholesterolaemia (FH) with LDL-C >130 mg/dL, uncontrolled stable atherosclerotic cardiovascular disease (ASCVD) with LDL-C >130 mg/dL or unstable ASCVD with LDL-C >100 mg/dL in combination with a statin and ezetimibe at maximum tolerated doses, and in patients who cannot tolerate or cannot be given statins with LDL-C >100 mg/dl.
Purpose Describe the efficacy and safety of PCSK9-I at a tertiary care hospital.
Material and methods Retrospective study was performed. We reviewed all cases treated with PCSK9-I from April 2016 to June 2017.
Demographic, clinical, analytical and treatment variables were collected at baseline and after the first follow-up visit (cut-off date 4 October 2017). These data were obtained from medical records.
Analysis was performed according to the intention-to-treat principle. The variables are presented by means and percentages. The Chi-square test was used for comparison among groups. Statistical analysis was performed using IBM® SPSS Statistics® v 22.0.
Results Up to the cut-off date, 38 patients (20 females) received a PCSK9-I. The median age was 56 years (range 35–80). In 19 cases a PCSK9-I was indicated for ASCVD, 15 for FH and four for both indications. Fifteen were statin intolerant and 7 ezetimibe intolerant. The mean baseline LDL-C level was 180.5±49.4 mg/dL.
PCSK9-I in combination with statins were prescribed in 25 patients (11 at maximum dose) and 24 with ezetimibe. Evolocumab was indicated in 27 cases and alirocumab in 11.
After the first follow-up visit (mean of 14.0±8.3 weeks), the mean LDL-C was 79.4±38.8 mg/dl, mean percent change, −56%; absolute change, −102.5 mg/dL. There were no significant differences in LDL-C reduction between evolocumab and alirocumab (−58% vs −50%; p=0.334). One patient had poor compliance due to adverse events (hair loss and nail fungus), although it is not described in the European Public Assessment Report (EPAR).
Conclusion LDL-C reductions obtained with PCSK9-I in clinical practice are similar to those described in clinical trials (50% to 70%).
PCSK9-I were well tolerated without discontinuations due to side-effects.
These new drugs bring a treatment opportunity to patients who are nowadays intolerant or non-responders to the currently available therapies.
No conflict of interest
Statistics from Altmetric.com
If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.