Lipid-efficacy RCT · N Engl J Med 2020
HAUSER-RCT trial
Evolocumab in Pediatric Heterozygous Familial Hypercholesterolemia.
The bottom line
In pediatric patients with heterozygous familial hypercholesterolemia already on lipid-lowering therapy, adding monthly evolocumab substantially lowered LDL cholesterol compared with placebo with a similar adverse event rate.
Primary lipid endpoint metUS status: US-approved
At a glance
- Medicine
- Evolocumab 420 mg monthly subcutaneous injections (104 patients)
- Compared with
- Placebo monthly subcutaneous injections (53 patients)
- People
- 157
- Who took part
- Patients 10 to 17 years of age with heterozygous familial hypercholesterolemia, on stable lipid-lowering treatment for at least 4 weeks before screening, with LDL cholesterol level of 130 mg per deciliter (3.4 mmol per liter) or more and a triglyceride level of 400 mg per deciliter (4.5 mmol per liter) or less
- Design
- 24-week, randomized, double-blind, placebo-controlled trial, 2:1 randomization
- Follow-up
- 24 weeks
- Main question
- Percent change in LDL cholesterol level from baseline to week 24
Main result
At week 24, the mean percent change from baseline in LDL cholesterol level was -44.5% in the evolocumab group and -6.2% in the placebo group, for a difference of -38.3 percentage points (P<0.001)
Effect on LDL cholesterol
Mean percent change from baseline: -44.5% (evolocumab) vs -6.2% (placebo), difference -38.3 percentage points (P<0.001); absolute change -77.5 mg per deciliter (-2.0 mmol per liter) vs -9.0 mg per deciliter (-0.2 mmol per liter), difference -68.6 mg per deciliter (-1.8 mmol per liter) (P<0.001)
Other results
Results for all secondary lipid variables were significantly better with evolocumab than with placebo.
Safety
The incidence of adverse events that occurred during the treatment period was similar in the evolocumab and placebo groups.
In the trial's own words
“At week 24, the mean percent change from baseline in LDL cholesterol level was -44.5% in the evolocumab group and -6.2% in the placebo group, for a difference of -38.3 percentage points (P<0.001). The absolute change in the LDL cholesterol level was -77.5 mg per deciliter (-2.0 mmol per liter) in the evolocumab group and -9.0 mg per deciliter (-0.2 mmol per liter) in the placebo group, for a difference of -68.6 mg per deciliter (-1.8 mmol per liter) (P<0.001).”
Quoted from the abstract of N Engl J Med 2020, checked word for word against PubMed. PubMed 32865373 · Full paper (DOI)
How this medicine works, where it stands and what it costs: Evolocumab (Repatha) and alirocumab (Praluent)
Common questions
What did the HAUSER-RCT trial find?
In pediatric patients with heterozygous familial hypercholesterolemia already on lipid-lowering therapy, adding monthly evolocumab substantially lowered LDL cholesterol compared with placebo with a similar adverse event rate.
Who was in HAUSER-RCT?
157 people. Patients 10 to 17 years of age with heterozygous familial hypercholesterolemia, on stable lipid-lowering treatment for at least 4 weeks before screening, with LDL cholesterol level of 130 mg per deciliter (3.4 mmol per liter) or more and a triglyceride level of 400 mg per deciliter (4.5 mmol per liter) or less.
What was HAUSER-RCT's main result?
At week 24, the mean percent change from baseline in LDL cholesterol level was -44.5% in the evolocumab group and -6.2% in the placebo group, for a difference of -38.3 percentage points (P<0.001)
How much did LDL cholesterol change in HAUSER-RCT?
Mean percent change from baseline: -44.5% (evolocumab) vs -6.2% (placebo), difference -38.3 percentage points (P<0.001); absolute change -77.5 mg per deciliter (-2.0 mmol per liter) vs -9.0 mg per deciliter (-0.2 mmol per liter), difference -68.6 mg per deciliter (-1.8 mmol per liter) (P<0.001)
Related trials
- ORION-10/11 (2020)Twice-yearly inclisiran injections lowered LDL cholesterol by about 50% compared with placebo in statin-treated patients with atherosclerotic cardiovascular disease, with mostly mild injection-site reactions as the main safety difference.
- ORION-9 (2020)In patients with heterozygous familial hypercholesterolemia, twice-yearly inclisiran injections significantly lowered LDL cholesterol compared with placebo, with a similar safety profile and a convenient infrequent dosing schedule.
- ODYSSEY OUTCOMES (2018)In post-ACS patients already on high-intensity statins, adding alirocumab reduced recurrent cardiovascular events and all-cause death compared with placebo.
- FOURIER-OLE (2022)Long-term evolocumab treatment after FOURIER maintained very low LDL-C with a persistently favorable safety profile over more than 8 years, and earlier initiation of evolocumab was associated with fewer cardiovascular events, including cardiovascular death, compared with delayed treatment start.
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