Novel insights into the management of German patients with very high cardiovascular risk eligible for PCSK9 inhibitor treatment: baseline characteristics from the PERI-DYS study

Background The reasons why patients are treated or not with PCSK9 inhibitors (PCSK9i) are incompletely understood. In Germany, access to PCSK9i is limited by local regulations and many high-risk cardiovascular patients do not receive these therapies. The PERI-DYS study aims to describe and compare two groups of dyslipidaemia patients at very high CV risk: those treated with PCSK9i compared with patients qualifying for but not treated with PCSK9i. Methods Observational study with up to 2000 consented patients, documented mainly by office-based cardiologists or physicians in lipid ambulances with data extracted from patient charts. Lipid lowering treatment (LLT) at enrolment includes ongoing PCSK9i use, newly initiated PCSK9i, statins, ezetimibe, and lipoprotein apheresis. Patients are followed for up to 3 years, with visits every 6±2 months to record LLT (drugs, dosing), other CV medications, lipid and glucose values, blood pressure, clinical events (major adverse cardiac and cerebrovascular events) and adverse drug reactions. Results As of 05 March 2021, 1488 patients have been enrolled across 70 sites. The majority of patients (91.5%) had heterozygous familial or non-familial hypercholesterolemia or mixed dyslipidaemia. At enrolment, 49.4% of patients were receiving PCSK9i (35.4% ongoing and 14.0% newly treated). Among PCSK9i users, the majority were receiving evolocumab 140 mg (n=567, 38.1% of all enrolled patients). There were no major differences in demographics and non-lipid lowering medication, with the exception of more females in the PCSK9i group. The estimated untreated LDL-C based on “back-calculation” was higher in patients who were on ongoing PCSK9i therapy than in those not on PCSK9i or newly treated with PCSK9i (Table 1). Physician-reported statin intolerance was much more common in the two PCSK9i groups compared with the non-PCSK9i group (67% versus 14%). Patients in the PCSK9i groups received fewer concomitant statins. Mean on-treatment total cholesterol and LDL-C were considerably lower in patients who were on ongoing PCSK9i compared to non-PCSK9i. Overall, nicotinic acid, fibrates, cholestagel, and omega-3 fatty acids were rarely used (data not shown). Conclusions Patients treated with PCSK9i and those qualifying for but not treated with PCSK9i had similar baseline characteristics, but the former had higher estimated untreated LDL-C values and a higher rate of statin intolerance. Ongoing follow-up will determine the prognostic importance of these findings. Funding Acknowledgement Type of funding sources: Private company. Main funding source(s): Amgen GmbH Germany