Evolocumab Treatment in Pediatric Patients With Homozygous Familial Hypercholesterolemia: Pooled Data From Three Open-Label Studies

CONCLUSIONS: In this pooled data analysis from 3 studies in pediatric patients with HoFH, evolocumab was well tolerated, with no new safety signals reported. These safety findings are consistent with findings from previous studies of evolocumab. Patients showed marked variability in LDL-C reduction. Results from this pooled analysis support guidelines suggesting a trial of PCSK9 inhibitor therapy regardless of estimated residual LDLR function.REGISTRATION: URL: https://www.clinicaltrials.gov; Unique identifier: NCT01624142, NCT03403374, and NCT02624869.PMID:38545781 | DOI:10.1161/ATVBAHA.123.320268

Source: Arteriosclerosis, Thrombosis and Vascular Biology - March 28, 2024 Category: Cardiology Authors: Frederick J Raal Robert A Hegele Andrea Ruzza J Antonio G L ópez Ajay K Bhatia Johnny Wu Huei Wang Daniel Gaudet Albert Wiegman Jian Wang Raul D Santos Source Type: research