New Trends and Therapies for Familial Hypercholesterolemia. In 4000 words. Please follow the following:
1-Quick introduction about FH : definition, Ho and HeFH , frequency, elevated risk of ASCVD(1, 2)
2-Impact of statin therapy and ezetimibe on LDL-C and ASCVD in both He and Ho FH (Quote impact on LDL-C and observational studies showing reduction in ASCVD ex- Versmissen et al
(4) in HeFH and in HoFH Raal
(6) 3-Say that despite this most FH patients persist with elevated LDL-C cholesterol
(7-9) 4-Current and specific new therapies PCSK9 inhibitors A-monoclonal antibodies (alirocumab and evolocumab)for HE and Ho FH
(10-12) B-small interference RNA (inclisiran)
(13) C-Oral PCSK9 I (Check if there is any publication already) Bempedoic acid for HEFH (no specific study but has patients with FH on the studies of patients with high LDL) ANGPTL3 inhibitors for Ho and refractory HeFH
(14, 15) MTP inhibitors (lomitapide Cuchel et al
(16) long term studies ex- Blom et al
(17). Lower registry
(18), CETP inhibitors specifically : Obicetrapib Genetic therapies CRISPR/Cas9 for HEFH Viral vectors for HoFH Trends: discuss restricted access in most parts of the world, high cost, need for risk stratification for greater cost effectiveness of therapies
(2, 19-23) discuss risk heterogeneity and how to identify higher risk FH patients (severe vs. resilient FH phenotypes)
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