New study highlights evolocumab’s potential in managing HoFH in Children

A pooled safety and efficacy analysis of data from 3 clinical studies demonstrated that evolocumab was well tolerated, with no new safety concerns identified for pediatric patients with homozygous familial hypercholesterolemia (HoFH). While there was variability in the reduction of LDL-C levels, a significant proportion of patients achieved meaningful reductions. The study suggests that evolocumab may be beneficial regardless of the level of residual LDL receptor activity.

The study, including patients aged 10 to 17 years across the TAUSSIG, RAMAN, and HAUSER-OLE trials, demonstrated encouraging results. Patients received either monthly or biweekly subcutaneous doses of evolocumab alongside standard statin therapy with or without ezetimibe. The primary endpoint focused on treatment-emergent adverse events, revealing upper respiratory tract infection, influenza, and acne as the most common, with a patient incidence rate of ≥5% per 100 patient-years.

Of the 39 patients analyzed, 79.5% harbored genotyped HoFH with LDLR pathogenic variants. Evolocumab exhibited good tolerability, with no new safety concerns identified. Serious treatment-emergent adverse events were reported in 13.3% of patients per 100 patient-years, showcasing a manageable safety profile consistent with previous findings on evolocumab.

Efficacy assessments centered on changes in lipid levels and PCSK9 from baseline to week 12. Although there was a modest median reduction (-2.9%) in LDL-C from baseline, a substantial portion (42.9%) achieved ≥15% reduction. Residual LDLR activity did not correlate with LDL-C reduction, suggesting potential benefits for patients irrespective of LDLR function.

Reference
Raal FJ, Hegele RA, Ruzza A, et al. Evolocumab Treatment in Pediatric Patients With Homozygous Familial Hypercholesterolemia: Pooled Data From Three Open-Label Studies. Arterioscler Thromb Vasc Biol. 2024;doi: 10.1161/ATVBAHA.123.320268. Epub ahead of print. PMID: 38545781.