Lipid-lowering Therapy with PCSK9 Inhibitors in the Real World Setting: Two-year Experience of a Regional Lipid Clinic

Background: PCSK9 inhibitors (PCSK9i) effectively lower cholesterol levels in randomized trials with reduction in cardiovascular outcomes and favorable safety profile. However, the access to PCSK9i is limited due to high cost and data regarding the use of PCSK9i in real-world practice is currently lacking.

Methods: Registry of all patients submitted for approval of PCSK9i (alirocumab or evolocumab) during the years 01.2016-11.2017 at a regional lipid clinic, outside of clinical trials. Patients` profile, approval rates, barriers to patient access, LDL-C reduction rates and adverse events were evaluated.

Results: Recommendation for PCSK9i treatment was given to 132 patients (120 complementary/private insurance, 10 compassionate programs and 2 within the "health basket"). Sixteen patients did not receive insurance approval. Additional 17 were approved but did not initiate therapy. All patients underwent dietary and exercise counseling. Of the 99 treated patients (47% females; mean age 61±11 years), half had probable/definite familial hypercholesterolemia (FH) (mean peak LDL-C level 305±88 vs. non-FH 204±39 mg/dl) and 64% had an established cardiovascular disease. Statin associated adverse effects were reported by 78%. Follow-up lipid panel was available in 64/99 patients. Mean LDL-C reduction was 59±18 percent (Figure) and 58% of the treated patients achieved LDL-C <70 mg/dl. Subjects with heterozygous FH had similar LDL-C decrease than those with non-FH (59±22% vs 60±14%, p=0.795). Side effects were reported by 10 patients: musculoskeletal complaints (4 patients) and flu-like symptoms (3) were the most frequent. Overall, 15 patients (15%) have discontinued treatment: 7 because of side effects, 5 due to end of compassionate program, 2 for unknown reasons and 1 because of nonresponse.

Conclusions: Patient selection by lipid clinic resulted in relatively high real-world PCSK9i insurance approval, with LDL-C lowering and safety comparable to published clinical trials. Cost and medication non-adherence are potential barriers to successful implementation of therapy and improved clinical outcomes.