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

共 50 条

[21] Diagnosis and treatment of hereditary angioedema
Canonica, G. W.
Rossi, O.
PANMINERVA MEDICA, 2012, 54 (03) : 241 - 253
[22] Life with Hereditary Angioedema: Then and Now
Lane, Charles Joseph
Grant, J. Andrew
Dougherty, Dane, III
POSTGRADUATE MEDICINE, 2012, 124 (01) : 144 - 151
[23] Hereditary Angioedema: Implications of Management
Pathria, Mohini
Krishnaswamy, Guha
Guarderas, Juan C.
SOUTHERN MEDICAL JOURNAL, 2017, 110 (02) : 101 - 106
[24] Updates on hereditary angioedema in pediatrics
El-Ghoneimy, Dalia H.
EGYPTIAN JOURNAL OF PEDIATRIC ALLERGY AND IMMUNOLOGY, 2019, 17 (02): : 51 - 60
[25] Hereditary angioedema with a focus on the child
Bennett, Gregory
Craig, Timothy
ALLERGY AND ASTHMA PROCEEDINGS, 2015, 36 (01) : 70 - 73
[26] Delayed Diagnosis of Hereditary Angioedema
Maskatia, Zahida Khan
Orson, Frank M.
AMERICAN JOURNAL OF THE MEDICAL SCIENCES, 2010, 340 (01) : 82 - 83
[27] Emerging Therapies in Hereditary Angioedema
Chen, Meng
Riedl, Marc A.
IMMUNOLOGY AND ALLERGY CLINICS OF NORTH AMERICA, 2017, 37 (03) : 585 - +
[28] The Role of Complement in Hereditary Angioedema
Levi, Marcel
Cohn, Danny M.
TRANSFUSION MEDICINE REVIEWS, 2019, 33 (04) : 243 - 247
[29] Hereditary Angioedema Management: Individualization
Epland, Kristin
Wayne, Melanie
Pein, Heather
JNP-JOURNAL FOR NURSE PRACTITIONERS, 2021, 17 (10): : 1199 - 1203
[30] Management of Hereditary Angioedema in Childhood
Wu, Eveline Y.
Frank, Michael M.
PEDIATRIC ALLERGY IMMUNOLOGY AND PULMONOLOGY, 2014, 27 (04) : 165 - 169

← 1 2 3 4 5 →