title: [Introduction of PCSK9 inhibitors : New perspectives in treatment and practical implementation].
abstract: Proprotein convertase subtilisin/kexin type 9 (PCSK9) is of critical importance in the regulation of the low-density lipoprotein (LDL) receptor-mediated metabolism of cholesterol. The discovery of mutations in the gene encoding PCSK9 in families with an autosomal dominant form of familial hypercholesterolemia (FH), which were later shown to be "gain-of-function" mutations, led to the development of antibodies against PCSK9. The efficacy in markedly reducing levels of LDL-cholesterol and preliminary evidence for benefits in the prevention of cardiovascular diseases indicated that special groups of patients can be more effectively treated. This includes forms of hypercholesterolemia refractory to conventional treatment as well as patients with FH and/or statin intolerance. Further information on long-term efficacy, tolerability and cost-effectiveness of PCSK9 inhibition and possibilities of implementation in the healthcare system are awaited from ongoing clinical outcome trials, such as FOURIER, ODYSSEY OUTCOMES, SPIRE 1 and 2 involving more than 70,000 high-risk patients.
authors.affiliation: Praxen Dres. T. Beckenbauer & S. Maierhof, Am Markt 11, 28195, Bremen, Deutschland. Klose.bremen@t-online.de.|Dres. I. van de Loo & K. Spieker, Gerold-Janssen-Str. 2a, 28359, Bremen, Deutschland. Klose.bremen@t-online.de.
authors.forename: G
authors.initials: G
authors.lastname: Klose
mesh_terms: D000911:Antibodies, Monoclonal / Q000008:administration & dosage* / Q000009:adverse effects; D000924:Anticholesteremic Agents / Q000008:administration & dosage* / Q000009:adverse effects; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D015195:Drug Design; D019317:Evidence-Based Medicine; D005858:Germany; D006801:Humans; D006937:Hypercholesterolemia / Q000175:diagnosis* / Q000188:drug therapy*; D058990:Molecular Targeted Therapy / Q000379:methods; D000091362:PCSK9 Inhibitors; D018579:Patient Selection; D017410:Practice Guidelines as Topic; D000071449:Proprotein Convertase 9 / Q000378:metabolism*; D016896:Treatment Outcome
chemical_list: D000911:Antibodies, Monoclonal; D000924:Anticholesteremic Agents; D000091362:PCSK9 Inhibitors; C472125:PCSK9 protein, human; D000071449:Proprotein Convertase 9
keywords: Cardiovascular diseases; Familial hypercholesterolemia; LDL cholesterol; Monoclonal antibodies; Proprotein convertase subtilisin/kexin type 9, human