Homozygous autosomal dominant hypercholesterolaemia
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Key result
Novel agents like mipomersen and lomitapide expand therapeutic options for extreme LDL-C in homozygous hypercholesterolemia.
Population
Patients with homozygous autosomal dominant hypercholesterolemia (hoADH)
Design
Review
Authors
Discussion
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Implication
May expand LDL-C lowering options in homozygous hypercholesterolemia; leaves open the need for outcome trials and long-term safety data.
Structured PICO
P
Population
Patients with homozygous autosomal dominant hypercholesterolemia (hoADH)
I
Intervention
Lipid-lowering therapies including bile acid sequestrants, statins, ezetimibe, lipoprotein apheresis, lomitapide, and mipomersen
This review summarizes the evolving therapeutic landscape for homozygous autosomal dominant hypercholesterolemia, including the introduction of novel LDL-C lowering agents like lomitapide and mipomersen.
Limitations
- Lack of large, randomized controlled clinical trials for lipoprotein apheresis due to the low incidence of hoADH.
- Safety concerns regarding hepatic fat accumulation with novel agents like lomitapide and mipomersen.
Cite This Study
Sjouke et al. (2015) conducted a review in Homozygous autosomal dominant hypercholesterolemia (hoADH). Lipid-lowering therapies (including lipoprotein apheresis, mipomersen, lomitapide) was evaluated. Novel lipid-altering pharmacological agents, such as mipomersen and lomitapide, along with lipoprotein apheresis, provide new therapeutic options for managing extremely elevated LDL-C levels in patients with homozygous autosomal dominant hypercholesterolemia.
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