Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema

被引：31

作者：

Cohn, Danny M. ^{[1
]}

Viney, Nicholas J. ^{[4
]}

Fijen, Laure M. ^{[1
]}

Schneider, Eugene ^{[4
]}

Alexander, Veronica J. ^{[4
]}

Xia, Shuting ^{[4
]}

Kaeser, Gwendolyn E. ^{[4
]}

Nanavati, Charvi ^{[4
]}

Baker, Brenda F. ^{[4
]}

Geary, Richard S. ^{[4
]}

Levi, Marcel ^{[5
,6
,7
]}

Meijers, Joost C. M. ^{[2
,3
]}

Stroes, Erik S. G. ^{[1
]}

机构：

[1] Univ Amsterdam, Amsterdam UMC, Dept Vasc Med, Amsterdam Cardiovasc Sci, Amsterdam, Netherlands

[2] Univ Amsterdam, Amsterdam UMC, Dept Expt Vasc Med, Amsterdam Cardiovasc Sci, Amsterdam, Netherlands

[3] Sanquin Res, Dept Mol & Cellular Hemostasis, Amsterdam, Netherlands

[4] Ionis Pharmaceut, Carlsbad, CA USA

[5] Univ Coll London Hosp NHS Fdn Trust, Dept Med, London, England

[6] Univ Coll London Hosp, Natl Inst Hlth Res, Cardiometab Programme, London, England

[7] UCL, Biomed Res Ctr, London, England

来源：

NEW ENGLAND JOURNAL OF MEDICINE | 2020年 / 383卷 / 13期

关键词：

PLASMA KALLIKREIN; DEFICIENCY;

D O I：

10.1056/NEJMoa1915035

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Hereditary angioedema is characterized by recurrent and unpredictable episodes of subcutaneous and mucosal swelling that can be life threatening. IONIS-PKK-L-Rx is a ligand-conjugated antisense oligonucleotide designed for receptor-mediated delivery to hepatocytes. In a compassionate-use pilot study, two patients with severe bradykinin-mediated angioedema were initially administered weekly subcutaneous injections of the unconjugated parent drug, IONIS-PKKRx, for 12 to 16 weeks, after which they received IONIS-PKK-L-Rx at a dose of 80 mg every 3 to 4 weeks for 7 to 8 months. Treatment was accompanied by a reduction in the angioedema attack rate.

引用

页码：1242 / 1247

页数：6

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