Prevention & lipidsResearch article
Evinacumab for Pediatric Patients With Homozygous Familial Hypercholesterolemia
- Single-arm open-label phase 3 trial
- part B
Authors' conclusion
Evinacumab constitutes a new treatment for pediatric patients with HoFH and inadequately controlled LDL-C despite optimized lipid-lowering therapy, lowering LDL-C levels by nearly half in these extremely high-risk and difficult-to-treat individuals.
Study context
- Population
- Children 5 to 11 years with genetically proven homozygous familial hypercholesterolemia (HoFH) and low-density lipoprotein cholesterol (LDL-C) >130 mg/dL despite optimized lipid-lowering therapy (incl. apheresis and lomitapide)
- Participants
- 14 children
Main finding
As reported in the abstract
LDL-C change from baseline · week 24
Evinacumab
−48.3%
mean · SE 10.4%
Further findings
ApoB, non-HDL cholesterol and total cholesterol, % change from baseline · week 24
- ApoB, mean [SE] % change
- −41.3% [9.0%]
- Non-HDL cholesterol, mean [SE] % change
- −48.9% [9.8%]
- Total cholesterol, mean [SE] % change
- −49.1% [8.1%]
- single-arm, open-label
Safety
Treatment-emergent adverse events
- Patients with treatment-emergent AEs
- 10 (71.4%)
- Patients with treatment-related events (nausea and abdominal pain)
- 2 (14.3%)
- Serious treatment-emergent AE (tonsillitis), not treatment-related
- n=1
Original publication
Evinacumab for Pediatric Patients With Homozygous Familial Hypercholesterolemia. Circulation. Published 20 October 2023. doi:10.1161/circulationaha.123.065529
Summarised from the published abstract; every number is checked against it