Effectiveness of alirocumab in treating pediatric patients with heterozygous familial hypercholesterolemia

According to a recent study, alirocumab may reduce low-density lipoprotein cholesterol (LDL-C) and other lipid parameters in pediatric patients with heterozygous familial hypercholesterolemia (HeFH) who have not achieved sufficient control with statins. This study’s findings were published in the journal, JAMA pediatrics.

In this phase 3, randomized clinical trial, 153 pediatric patients aged 8-17 years with HeFH, LDL-C levels of 130 mg/dL or higher, and undergoing statin or other lipid-lowering therapy (LLT) were randomly assigned to receive subcutaneous alirocumab or a placebo and dosed every 2 weeks (Q2W) or every 4 weeks (Q4W). The dosage depended on weight (if <50 kg then 40 mg for Q2W or 150 mg for Q4W; if ≥50 kg then 75 mg for Q2W or 300 mg for Q4W) and was adjusted at week 12 if LDL-C was 110 mg/dL or higher at week 8. Following the 24-week double-blind phase, patients could continue alirocumab treatment in an 80-week open-label phase. The primary endpoint of the study was the percentage change in LDL-C from baseline to week 24 in each group.

Alirocumab demonstrated statistically significant decreases in LDL-C compared to the placebo in both groups at week 24. The least squares mean difference in percentage change from baseline was -43.3% for the Q2W group and -33.8% for the Q4W group. A hierarchical analysis of secondary efficacy endpoints revealed notable enhancements in other lipid parameters at weeks 12 and 24 with alirocumab. The findings from the open-label period were consistent with those from the double-blind period.

Based on the above results, it can be concluded that alirocumab dosed every two weeks or every four weeks may lower LDL-C levels and improve other lipid parameters in pediatric patients with HeFH who have not achieved adequate control with statins.