1. RUTHERFORD-2
Trial design: Patients with heterogeneous familial hypercholesterolemia (HeFH) on statins were randomized in a 2:2:1:1 fashion to subcutaneous evolocumab 140 mg q2 weeks, 420 mg every month, placebo q2 weeks or placebo every month, and followed for 12 weeks.
Results
(p < )
Evolocumab 140 mg vs. placebo: mean LDL 64.6 vs mg/dl (59.2% absolute reduction, p < 0.001); change from baseline for ApoB: -49.8% vs. -0.7%, p < , HDL: 8.1% vs. -1.2%, p <
Evolocumab 420 mg vs. placebo: mean LDL 68.4 vs mg/dl (61.3% absolute reduction, p < 0.001); change from baseline for ApoB: -44.8% vs. 4.6%, p < , HDL: 5.4% vs. -3.7%, p =
100
68.0
63.0 % 50
Conclusions
Treatment of HeFH patients already on statins with evolocumab 140 mg q2 weeks or 420 mg every month resulted in ~60% ↓ in LDL levels at 12 weeks compared with placebo
Adds to the evolving body of literature with PCSK9 inhibitors such as evolocumab; CV outcomes trials and longer-term follow-up data are awaited
2.0
2.0
Proportion with LDL <70 mg/dl at week 12
Evolocumab 140 mg q2w
n = 111
Placebo q2w
n = 55
Placebo q1m
n = 55
Evolocumab 420 mg q1m
n = 110
Raal FJ, et al. Lancet 2014;Oct 1:[Epub]