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The DOI matches its Crossref record; 4 of the 4 references it cites were found in a registry or an index.

Effect of the Proprotein Convertase Subtilisin/Kexin 9 Monoclonal Antibody, AMG 145, in Homozygous Familial Hypercholesterolemia

Stein, Evan A., Honarpour, Narimon, Wasserman, Scott M., Xu, Feng, Scott, Rob, Raal, Frederick J.·2013·Circulation
DOI 10.1161/circulationaha.113.004678
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Abstract

Background—: Homozygous familial hypercholesterolemia is a rare, serious disorder with a substantial reduction in low-density lipoprotein (LDL) receptor function, severely elevated LDL cholesterol, cardiovascular disease, and often death in childhood. Response to conventional drug therapies is modest. Monoclonal antibodies to proprotein convertase subtilisin/kexin 9 (PCSK9) reduce LDL cholesterol in heterozygous familial hypercholesterolemia. The effect in homozygous familial hypercholesterolemia is unknown and uncertain. We evaluated the efficacy and safety of AMG 145 in an open-label, single-arm, multicenter, dose-scheduling pilot study in patients with homozygous familial hypercholesterolemia. Methods and Results—: Eight patients with LDL receptor–negative or –defective homozygous familial hypercholesterolemia on stable drug therapy were treated with subcutaneous 420 mg AMG 145 every 4 weeks for ≥12 weeks, followed by 420 mg AMG 145 every 2 weeks for an additional 12 weeks. All patients completed both treatment periods. Mean change from baseline in LDL cholesterol at week 12 was −16.5% (range, 5.2% to −43.6%; P =0.0781) and −13.9% (range, 39.9% to −43.3%; P =0.1484) with 4- and 2-week dosing, respectively. No reduction was seen in the 2 receptor-negative patients. Over the treatment periods, mean±SD LDL cholesterol reductions in the 6 LDL receptor–defective patients were 19.3±16% and 26.3±20% with 4- and 2-week dosing, respectively ( P =0.0313 for both values), ranging from 4% to 48% with 2-week dosing. No serious side effects were reported. Conclusion—: This study demonstrates significant and dose-related LDL cholesterol lowering with a PCSK9 monoclonal antibody in homozygous familial hypercholesterolemia patients with defective LDL receptor activity but no reduction in those who were receptor negative. Clinical Trial Registration—: URL: http://www.clinicaltrials.gov . Unique identifiers: NCT01588496 and NCT01624142.

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Crossref · 10.1161/circulationaha.113.004678

Cites

4 of 4 references verified0 citation errors · 0 not checkable

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Citations

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Research works printed but not linked: 23

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Metadata

Authors
Stein, E. A., Honarpour, N., Wasserman, S. M., Xu, F., Scott, R., & Raal, F. J.
Affiliations
From the Metabolic and Atherosclerosis Research Center, Cincinnati, OH (E.A.S.); Global Development (N.H., S.M.W., R.S.) and Biostatistics (F.X.), Amgen Inc, Thousand Oaks, CA; and Carbohydrate & Lipid Metabolism Research Unit, Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa (F.J.R.).
Venue
Circulation
Publisher
Ovid Technologies (Wolters Kluwer Health)
Published
2013-11-05
Volume
128
Issue
19
Pages
2113-2120
ISSN
  • 0009-7322
  • 1524-4539
DOI
10.1161/circulationaha.113.004678
Cited by
300
References
4
Language
en

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