Key result
Rosuvastatin drives ~18-point greater LDL-C reduction vs ezetimibe in pediatric familial hypercholesterolemia.
Why the study?
Individuals with heterozygous familial hypercholesterolemia require early lipid-lowering therapy, but comparative data on rosuvastatin versus ezetimibe and their effects on arterial injury markers in children are limited.
Does rosuvastatin improve LDL-C reduction and non-invasive arterial injury markers compared to ezetimibe in children with heterozygous familial hypercholesterolemia?
RCT (n=45)
Open-label
Simple randomization
No
Does rosuvastatin improve LDL-C reduction and non-invasive arterial injury markers compared to ezetimibe in children with heterozygous familial hypercholesterolemia?
Absolute Event Rate: 36.7% vs 18.3%
p-value: p=<0.001
Rosuvastatin 5 mg daily provides superior short-term LDL-C lowering efficacy compared to ezetimibe 10 mg daily in pediatric patients with familial hypercholesterolemia, with equivalent short-term safety.
Supports rosuvastatin preference over ezetimibe for LDL-C lowering in pediatric HeFH; extends direct RCT comparative data in children.
Background and aims: Individuals with familial hypercholesterolemia (FH) require early lipid-lowering therapy (LLT). We compared rosuvastatin versus ezetimibe treatment and the role of major non-invasive arterial injury markers in children with heterozygous FH.Methods: In this prospective, open-label, randomized study, 45 children with FH [median age 10.5 years (IQR 8.7–14.5)] received rosuvastatin 5 mg OD or ezetimibe 10 mg OD for 6.2 months. Carotid intima-media thickness (cIMT), reactive hyperemia index (RHI), pulse wave velocity (PWV), and beta stiffness were evaluated in relation to anthropometric and lipid-related variables.Results: Rosuvastatin produced a greater LDL-C reduction than ezetimibe (36.7% vs. 18.3%; p < 0.001) with 9/20 (45%) children achieving LDL-C <3.5 mmol/L compared to 2/15 (13%) in ezetimibe group. No reports of myalgias, elevations in transaminases, or creatine kinase (CK) were observed in any of the groups. Markers at baseline correlated mainly with age and anthropometry. Beta stiffness decreased during LLT in the overall cohort (−0.24; 95% CI −0.51 to −0.03, p = 0.002) and in the rosuvastatin group (−0.4; 95% CI −0.7 to −0.1, p = 0.028), with no between-group difference (p = 0.266). This likely reflects regression to the mean, as LDL-C reduction and treatment allocation had only small effects across all markers.Conclusions: Rosuvastatin achieved greater LDL-C reduction than ezetimibe in children with FH, without difference in safety and tolerability profiles. Arterial injury markers correlated mainly with age and anthropometry, and their short-term changes were not treatment-related, likely due to limited power and the short follow-up.
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Skarlovnik et al. (2026) conducted an RCT in Heterozygous familial hypercholesterolemia (n=45). Rosuvastatin vs. Ezetimibe 10 mg once daily was evaluated on Percentage reduction in LDL-C from baseline (p=<0.001). Rosuvastatin 5 mg daily produced a significantly greater LDL-C reduction compared to ezetimibe 10 mg daily (36.7% vs. 18.3%; p < 0.001) in children with familial hypercholesterolemia.
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