Cognitive function with evolocumab in pediatric heterozygous familial hypercholesterolemia

• DOI: 10.1016/j.jacl.2022.07.005
• Publication Date: 2022-07-21T06:45:43Z
• Authors: Daniel Gaudet; Andrea Ruzza; Ian Bridges; Paul Maruff; Adrian Schembri; Andrew Hamer; François Mach; Jean Bergeron; Isabelle Gaudet; Julie St Pierre; John J.P. Kastelein; G. Kees Hovingh; Albert Wiegman; Frederick J. Raal; Raul D. Santos
• Keywords: Evolocumab; Cognitive test; Tolerability

Evolocumab is a fully human monoclonal antibody inhibitor of PCSK9 approved for lowering low-density lipoprotein cholesterol in adults and pediatric patients with familial hypercholesterolemia (FH). The cognitive safety evolocumab has been established but not yet described patients.To determine the effects on function heterozygous FH.Cognitive was assessed during 24-week, randomized, double-blind, placebo-controlled study (HAUSER-RCT) evaluating efficacy, safety, tolerability 24 weeks monthly subcutaneous injections FH. Cognitive endpoints included changes from baseline to week test scores domains psychomotor function, attention, visual learning, executive function. Between-group differences age-standardized mean score were analyzed using analysis covariance models point estimates 95% confidence interval (CI). Magnitudes difference between treatment groups (Cohen's d) reliable change indices calculated each test.At 24, similar groups. Differences (95% CI) placebo Groton Maze Learning, One-Card Identification, Detection tests 0.1 (-0.2, 0.4), -0.1 (-0.5, 0.3 (0.0, 0.7), (-0.1, 0.8), respectively. For all tests, abnormal clinically important decline occurred lesser frequency group.In FH, 24-week did negatively influence cognition.This funded designed by Amgen.