Background:
Evolocumab is the first
monoclonal antibody against
proprotein convertase subtilisin/kexin type 9 (PCSK9) approved in Japan for the treatment of patients with
familial hypercholesterolemia (FH) and
hypercholesterolemia (HC). This study assessed the 12-week effectiveness and safety of
low-density lipoprotein cholesterol (
LDL-C)-lowering
therapy by PCSK9 inhibition in patients with FH (homozygous [
HoFH] or heterozygous [HeFH]) and HC by analyzing
evolocumab data collected in the real-world setting in Japan. Methods and Results: Overall, 427 patients (mean±SD age, 61.6±13.8 years; female, 38.4%; 28
HoFH, 320 HeFH, 79 HC), enrolled from 299 clinical sites, were included in the safety analysis set. The major cardiovascular risk factors were
coronary artery disease (77.3%),
diabetes mellitus/
impaired glucose tolerance (38.6%), and
hypertension (65.1%). Median follow-up duration was 85.0 days. After 12 weeks of
evolocumab treatment, the mean±SD percent change from baseline in
LDL-C was -45.5%±27.0% (n=23) in
HoFH (P<0.001 vs. baseline; t-test), -54.2%±29.0% (n=280) in HeFH (P<0.001), and -64.6%±22.4% (n=72) in HC (P<0.001) patients. The incidence of
adverse drug reactions was 5.4% (23/427). Conclusions: Results suggest that patients receiving
evolocumab treatment in the real-world setting were predominantly those with FH and HC in the
secondary prevention group.
LDL-C-lowering effectiveness with
evolocumab was observed in FH (both
HoFH and HeFH) and HC patients.