Abstract
Statins are indicated for treatment of patients with heterozygous familial hypercholesterolaemia: these drugs inhibit hepatic cholesterol synthesis and indirectly raise transcription and expression of low-density lipoprotein (LDL) receptors, resulting in augmented residual LDL receptor function. Unfortunately, estimates of the effectiveness of statins in this setting are missing, since placebo-controlled trials in patients with familial hypercholesterolaemia have not been done. Observational evidence about statin treatment in a UK cohort 1 Neil A Cooper J Betteridge J et al. Reductions in all-cause, cancer, and coronary mortality in statin-treated patients with heterozygous familial hypercholesterolaemia: a prospective registry study. Eur Heart J. 2008; 29: 2625-2633 Crossref PubMed Scopus (340) Google Scholar and a Dutch follow-up study 2 Versmissen J Oosterveer DM Yazdanpanah M et al. Efficacy of statins in familial hypercholesterolaemia: a long term cohort study. BMJ. 2008; 337: a2423 Crossref PubMed Scopus (528) Google Scholar provides the best available support for a substantially improved outcome in asymptomatic patients with familial hypercholesterolaemia. In agreement with this, patients with familial hypercholesterolaemia in the ENHANCE study, 3 Kastelein JJ Akdim F Stroes ES et al. Simvastatin with or without ezetimibe in familial hypercholesterolemia. N Engl J Med. 2008; 358: 1431-1443 Crossref PubMed Scopus (1111) Google Scholar who had been treated with statins intensively, had a normal mean baseline carotid intima-media thickness. Effect of a monoclonal antibody to PCSK9, REGN727/SAR236553, to reduce low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolaemia on stable statin dose with or without ezetimibe therapy: a phase 2 randomised controlled trialREGN727 was well tolerated and achieved substantial further LDL-C reduction in patients with heterozygous familial hypercholesterolaemia and elevated LDL-C treated with high-dose statins, with or without ezetimibe. REGN727 has the potential to provide optimum control of LDL-C in patients with this disorder. Full-Text PDF
Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
