Effectiveness and safety of proprotein convertase subtilisin/kexin type 9 inhibitors in patients with familial hypercholesterolemia. Our experience in implementing the drug program of the Polish National Health Fund

Introduction: Heterozygous familial hypercholesterolemia (FH) is characterized by an elevated plasma low-density lipoprotein cholesterol (LDL-C) concentration despite intensive statin and ezetimibe therapy, which significantly increases the cardiovascular risk. Aim: The study evaluated the efficacy and safety of proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors, alirocumab and evolocumab, in reducing lipids in patients with FH. Material and methods: This was a single-center analysis of 22 patients diagnosed with FH treated with the PCSK9 inhibitors under the drug program of the National Health Fund. The follow-up interviews and laboratory tests were performed at baseline (22 patients), 3 months (22 patients) and 15 months (9 patients) after the first dose of PCSK9 inhibitors. Results: The mean (SD) baseline level of the total LDL-C fraction was 4.7 +/- 1.6 mmol/l in the whole group of patients and was significantly reduced after 3 and 15 months of PCSK9 inhibitors therapy to 1.7 +/- 1.6 and 1.6 +/- 1.1 mmol/l, respectively. The average percentage of reduction in LDL-C level was 64.9 +/- 23.7% after 3 months and 66.9 +/- 18.4% after 15 months. In comparison with base-line, a significant reduction in total cholesterol was observed at both time points (p < 0.0002). There were no adverse cardiovascular events or significant growth in the level of alanine transaminase, creatinine, and creatine kinase throughout the study. Conclusions: Patients with FH treated with PCSK9 inhibitors achieved a significant reduction of LDL-C and total cholesterol with the safety of this treatment in follow-up.