In questa valutazione in real life, l'aggiunta di anticorpi monoclonali PCSK9 alla terapia standard di pazienti ad alto rischio cardiovascolare portava a una marcata riduzione del colesterolo LDL, di quasi il 60%, simile a quella osservata in trial clinici randomizzati. I soggetti FH eterozigoti hanno mostrato una riduzione di LDL-C simile a quella dei soggetti non FH. L’obiettivo di LDL-C<100 mg/dL è stato raggiunto dal 76%, quello di LDL-C<70 mg/dL dal 58% e LDL-C<40 mg/dL dal 18%. Gli effetti indesiderati sono stati riportati dal 10% degli individui, principalmente disturbi muscolo-scheletrici e sintomi simil-influenzali, mentre il trattamento è stato interrotto nel 15% dei casi.
AIM: 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 limited.
METHODS: Data on all patients submitted for approval of PCSK9i at a regional lipid clinic, outside of clinical trials. Patients' profile, approval rates, low-density lipoprotein cholesterol (LDL-C) reduction rates, and adverse events were evaluated.
RESULTS: Recommendation for PCSK9i was given to 133 patients; 16 did not receive insurance approval and additional 16 were approved but did not initiate therapy. Of the 101 treated patients (47% females; mean age 61 ± 11 years), 52 had probable/definite familial hypercholesterolemia (FH) (peak LDL-C level 305 ± 87 mg/dL vs non-FH 204 ± 39 mg/dL) and 62% had an established cardiovascular disease. Statin intolerance was reported by 77%. Follow-up lipid panel was available in 66/101 patients: mean LDL-C reduction was 59% ± 19. Subjects with heterozygous FH had similar LDL-C decrease than those with non-FH (59% ± 22 vs 60% ± 14, P = .792). LDL-C < 100 mg/dL was achieved by 76%, LDL-C < 70 mg/dL by 58% and LDL-C < 40 mg/dL by 18% of those with follow-up data. Side effects were reported by 10%, mainly musculoskeletal complaints and flu-like symptoms, and 15% have discontinued treatment.
CONCLUSIONS: Patient selection by a regional lipid clinic resulted in a high real-world PCSK9i insurance approval, with efficacy and safety comparable to randomized clinical trials. Cost and medication nonadherence are potential barriers to successful implementation of therapy in routine clinical care.