The Long-Term Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
Authors' conclusion
In patients with HoFH, evinacumab demonstrated substantial and sustained LDL-C reduction regardless of LDLR function, and was generally well tolerated.
Study context
- Population
- Patients with HoFH on stable lipid-lowering therapies ± lipoprotein apheresis, screening low-density lipoprotein cholesterol (LDL-C) ≥70 mg/dL, who completed the 24-week double-blind period
- Participants
- 64 patients
- Outcome
- Mean LDL-C reduction from baseline
- Follow-up
- Week 48 (end of OLTP)
Methodological detailssingle-group, descriptive
- Single-group
- Descriptive
Main finding
As reported in the abstract
| Group | Value | Mean reduction | Baseline LDL-C |
|---|---|---|---|
| Evinacumab | mean reduction 46.3% | 134.3 ± 117.3 mg/dL | 250.5 ± 162.3 mg/dL |
Evinacumab
Valuemean reduction 46.3%
Mean reduction134.3 ± 117.3 mg/dL
Baseline LDL-C250.5 ± 162.3 mg/dL
Further findings
Mean reduction
| Outcome | Null-null | Non-null |
|---|---|---|
| Mean LDL-C reduction by LDLR variant (subgroup) · week 48 | 47.2% | 45.9% |
Safety
| Outcome | Values |
|---|---|
| Adverse events | Evinacumab 73.4% |
| Evinacumab-related adverse events (drug hypersensitivity, infusion-related reaction and asthenia, generalized pruritis, muscle spasms) | Evinacumab 6.3% |
Original publication
The Long-Term Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia. JACC Adv. Published 11 October 2023. doi:10.
Summarised from the published abstract; every number is checked against it