Evolocumab in Pediatric Heterozygous Familial Hypercholesterolemia Patients - HAUSER-RCT

Oct 01, 2020
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Author/Summarized by Author:
Anthony A. Bavry, MD,MPH,FACC
Summary Reviewer:
Deepak L. Bhatt, MD, MPH, MBA, FACC
Trial Sponsor:
Amgen
Date Presented:
08/29/2020
Date Published:
09/30/2020
Date Updated:
10/01/2020
Original Posted Date:
08/29/2020
References

Contribution To Literature:

The HAUSER-RCT trial showed that evolocumab was superior to placebo at reducing LDL-C. 

Description:

The goal of the trial was to evaluate evolocumab compared with placebo among pediatric patients with heterozygous familial hypercholesterolemia.


Study Design

  • Randomized
  • Parallel
  • Placebo

Eligible pediatric patients with heterozygous familial hypercholesterolemia were randomized to evolocumab 420 mg subcutaneous injection monthly (n = 104) versus placebo (n = 53) for 24 weeks.

  • Total number of enrollees: 157
  • Duration of follow-up: 24 weeks
  • Mean patient age: 13.7 years
  • Percentage female: 59%
  • Percentage with diabetes: 1%

Inclusion criteria:

  • Patients 10-17 years of age
  • Received stable lipid-lowering treatment for at least 4 weeks before screening
  • Low-density lipoprotein cholesterol (LDL-C) ≥130 mg/dl and triglycerides ≤400 mg/dl

Principal Findings:

The primary outcome, percent change in LDL-C from baseline to 24 weeks, was -44.5% in the evolocumab group compared with -6.2% in the placebo group (p < 0.001).

Secondary outcomes:

  • Any adverse event: 62% of evolocumab group compared with 64% of the placebo group (p = nonsignificant)

Interpretation:

Among pediatric patients with heterozygous familial hypercholesterolemia, evolocumab was associated with a significant reduction in LDL-C compared with placebo. Adverse events were similar between treatment groups. Further studies powered for clinical outcomes are warranted.

References:

Santos RD, Ruzza A, Hovingh K, et al., on behalf of the HAUSER-RCT Investigators. Evolocumab in Pediatric Heterozygous Familial Hypercholesterolemia. N Engl J Med 2020;383:1317-27.

Editorial: de Ferranti SD. Evolocumab in Children With Heterozygous Familial Hypercholesterolemia. N Engl J Med 2020;383:1385-6.

Presented by Dr. Raul Santos at the European Society of Cardiology Virtual Congress, August 29, 2020.

Clinical Topics: Diabetes and Cardiometabolic Disease, Dyslipidemia, Prevention, Hypertriglyceridemia, Lipid Metabolism, Nonstatins, Primary Hyperlipidemia

Keywords: ESC Congress, ESC20, Cholesterol, LDL, Dyslipidemias, Hyperlipoproteinemia Type II, Metabolic Syndrome, Pediatrics, Primary Prevention, Triglycerides


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