Long-term PCSK9 inhibitor safe and effective in HeFH
Long-term Safety, Tolerability, and Efficacy of Evolocumab in Patients With Heterozygous Familial HypercholesterolemiaLiterature - Hovingh GK, Raal FJ, Dent R, et al. - J Clin Lipidol (2017), doi: 10.1016/j.jacl.2017.09.003
- 281 patients in the evolocumab plus SOC group completed the OLE program and 144 in the SOC alone group, resulting in discontinuation rates of 2.8% and 4.6%, respectively.
- Patients who received evolocumab plus SOC for 48 weeks showed a mean reduction of 53.5% in calculated LDL-C levels (mean: -2.1 [SD: 0.07] mmol/L) compared to the levels at baseline of the parent study. The group with SOC alone had an increase of 2.1% after 48 weeks (0.03 [0.09] mmol/L).
- Similar patterns were observed for other lipid parameters; a decrease for apolipoprotein B, non-high-density lipoprotein-C , triglycerides and lipoprotein(a) and an increase in HDL-C in patients who received evolocumab plus SOC.
- Adverse event (AE) rates for the group of evolocumab plus SOC and SOC alone were 79.9% and 66.9%, respectively, without any permanent discontinuation in both groups due to AE.
- Infections and infestations (47.8% vs 37.1%), musculoskeletal and connective tissue disorders (33.2% vs. 21.9%), general disorders and administration site conditions (25.3% vs. 7.3%), gastrointestinal disorders (19.7% vs. 12.6%) and nervous system disorders (14.5% vs. 7.9%) were more common in the evolocumab plus SOC group compared to the SOC alone group.
- No differences in the number of serious AE was observed between the two groups.
This open-label extension trial of evolocumab demonstrated that long-term use (48 weeks) of this PCSK9 inhibitor in combination with SOC reduced the levels of LDL-C and was well tolerated in a HeFH patient population of , who need additional therapy to reduce their CV risk.