Administration of gene therapy for haemophilia - The hub and spoke model and its regional differences and challenges

被引：5

作者：

Miesbach, Wolfgang ^{[1
]}

Boban, Ana ^{[2
]}

Chowdary, Pratima ^{[3
]}

Coppens, Michiel ^{[4
,5
]}

Crato, Miguel ^{[6
]}

Jimenez-Yuste, Victor ^{[7
]}

Klamroth, Robert ^{[8
]}

Makris, Mike ^{[9
]}

Mulders, Greta ^{[10
]}

Peyvandi, Flora ^{[11
,12
,13
]}

机构：

[1] Univ Hosp Frankfurt, Inst Transfus Med, Med Clin 2, Frankfurt, Germany

[2] Univ Zagreb, Univ Hosp Ctr Zagreb, Sch Med, Haemophilia Ctr,Dept Haematol, Zagreb, Croatia

[3] Royal Free Hosp, Katharine Dormandy Haemophilia & Thrombosis Ctr, London, England

[4] Locat Univ Amsterdam, Amsterdam UMC, Hemophilia Treatment Ctr, Dept Vasc Med, Amsterdam, Netherlands

[5] Amsterdam Cardiovasc Sci, Pulm Hypertens & Thrombosis, Amsterdam, Netherlands

[6] European Haemopilia Consortium, Brussels, Belgium

[7] Univ Autonoma Madrid, Hosp Univ La Paz IdiPaz, Hematol Dept, Madrid, Spain

[8] Vivantes Hosp Friedrichshain, Haemophilia Treatment Ctr, Dept Internal Med & Vasc Med, Berlin, Germany

[9] Univ Sheffield, Dept Infect Immun & Cardiovasc Dis, Sheffield, England

[10] Erasmus MC, Dept Hematol, Rotterdam, Netherlands

[11] Fdn IRCCS Ca Granda Osped Maggiore Policlin, Angelo Bianchi Bonomi Hemophilia & Thrombosis Ctr, Milan, Italy

[12] Fdn Luigi Villa, Milan, Italy

[13] Univ Milan, Dept Pathophysiol & Transplantat, Milan, Italy

来源：

HAEMOPHILIA | 2024年 / 30卷 / 03期

关键词：

D O I：

10.1111/hae.14985

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：855 / 857

页数：3

共 4 条

[1] Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues [J].

Leebeek, Frank W. G. ;

Miesbach, Wolfgang .

BLOOD, 2021, 138 (11) :923-931

[2] Gene therapy of hemophilia: Hub centres should be haemophilia centres: A joint publication of EAHAD and EHC [J].

Miesbach, Wolfgang ;

Baghaei, Fariba ;

Boban, Ana ;

Chowdary, Pratima ;

Coppens, Michiel ;

Hart, Daniel P. ;

Jimenez-Yuste, Victor ;

Klamroth, Robert ;

Makris, Mike ;

Noone, Declan ;

Peyvandi, Flora .

HAEMOPHILIA, 2022, 28 (03) :E86-E88

[3] Delivery of AAV-based gene therapy through haemophilia centres-A need for re-evaluation of infrastructure and comprehensive care: A Joint publication of EAHAD and EHC [J].

Miesbach, Wolfgang ;

Chowdary, Pratima ;

Coppens, Michiel ;

Hart, Daniel P. ;

Jimenez-Yuste, Victor ;

Klamroth, Robert ;

Makris, Michael ;

Noone, Declan ;

Peyvandi, Flora .

HAEMOPHILIA, 2021, 27 (06) :967-973

[4] Haemophilia gene therapy-Update on new country initiatives [J].

Reiss, Ulrike M. ;

Mahlangu, Johnny ;

Ohmori, Tsukasa ;

Ozelo, Margareth C. ;

Srivastava, Alok ;

Zhang, Lei .

HAEMOPHILIA, 2022, 28 :61-67

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