Article Text
Abstract
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