Prevention & lipidsMeta-analysis
Current treatments for the management of homozygous familial hypercholesterolaemia: a systematic review and commentary
- Systematic review (mixed-methods) of primary interventional studies
- no meta-analysis (data heterogeneous)
Authors' conclusion
In practice, multiple treatments are required to treat HoFH.
Study context
- Population
- Homozygous familial hypercholesterolaemia (HoFH)26 studies (4 randomized controlled trials (RCTs))
- Comparison
- Ezetimibe, PCSK9i, lomitapide, evinacumab, with or without LDL apheresis
- Outcome
- LDL-C reduction
Pooled result
As reported in the abstract
- PCSK9i safe and moderately effective (RCTs at low risk of bias)
- Evinacumab safe and effective in all HoFH subgroups (randomized controlled trial (RCT))
- Lomitapide efficacious in a single-arm trial; issues with adverse events, tolerability and adherence
- Ezetimibe moderately effective when combined with a statin (RCT)
- LDL apheresis reported effective; evidence at very high risk of bias
- All interventions lowered low-density lipoprotein cholesterol (LDL-C); magnitude and certainty of evidence varied
- data heterogeneous, not suitable for meta-analyses
Safety
Lomitapide adverse events, tolerability and adherence
- Issues with adverse events, tolerability and adherence identified with lomitapide
Original publication
Current treatments for the management of homozygous familial hypercholesterolaemia: a systematic review and commentary. Eur J Prev Cardiol. Published 18 April 2024. doi:10.1093/eurjpc/zwae144
Summarised from the published abstract; every number is checked against it