How I approach: Previously untreated patients with severe congenital hemophilia A

被引：1

作者：

Thornburg, Courtney D. ^{[1
,2
]}

机构：

[1] Rady Childrens Hosp San Diego, San Diego, CA 92123 USA

[2] Univ Calif San Diego, La Jolla, CA USA

来源：

PEDIATRIC BLOOD & CANCER | 2018年 / 65卷 / 12期

关键词：

factor replacement; genotyping; hemophilia; inhibitor; prophylaxis; IMMUNOLOGICAL DANGER SIGNALS; INHIBITOR DEVELOPMENT; FACTOR-VIII; EMICIZUMAB PROPHYLAXIS; LIFE; PREVENTION; MANAGEMENT; TOLERANCE; OUTCOMES; THERAPY;

D O I：

10.1002/pbc.27466

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Previously untreated patients with severe hemophilia A are a vulnerable population at risk for severe bleeding which is currently managed with exogenous clotting factor replacement. The primary burden of current treatment is high-titer inhibitor development. Evolving data on current treatment products as well as emerging therapeutics may inform treatment decisions to prevent bleeding and inhibitor formation. Considerations for diagnosis, education, and shared decision-making related to product choice and treatment regimen are discussed.

引用

页数：7

共 55 条

[1] Developing a two-sided intervention to facilitate shared decision-making in haemophilia: decision boxes for clinicians and patient decision aids for patients [J].

Athale, A. ;

Giguere, A. ;

Barbara, A. ;

Krassova, S. ;

Iorio, A. .

HAEMOPHILIA, 2014, 20 (06) :800-806

[2] The EPIC study: a lesson to learn [J].

Auerswald, G. ;

Kurnik, K. ;

Aledort, L. M. ;

Chehadeh, H. ;

Loew-Baselli, A. ;

Steinitz, K. ;

Reininger, A. J. .

HAEMOPHILIA, 2015, 21 (05) :622-628

[3] Early prophylaxis/FVIII tolerization regimen that avoids immunological danger signals is still effective in minimizing FVIII inhibitor developments in previously untreated patients - long-term follow-up and continuing experience [J].

Auerswald, G. ;

Bidlingmaier, C. ;

Kurnik, K. .

HAEMOPHILIA, 2012, 18 (01) :E18-E20

[4] Definitions in hemophilia: communication from the SSC of the ISTH [J].

Blanchette, V. S. ;

Key, N. S. ;

Ljung, L. R. ;

Manco-Johnson, M. J. ;

Van Den Berg, H. M. ;

Srivastava, A. .

JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 2014, 12 (11) :1935-1939

[5] Novel therapeutics for hemophilia and other bleeding disorders [J].

Callaghan, Michael U. ;

Sidonio, Robert ;

Pipe, Steven W. .

BLOOD, 2018, 132 (01) :23-30

[6]

Chai-Adisaksopha C, 2017, COCHRANE DB SYST REV, V9

[7] Guideline on the management of haemophilia in the fetus and neonate [J].

Chalmers, Elizabeth ;

Williams, Michael ;

Brennand, Janet ;

Liesner, Ri ;

Collins, Peter ;

Richards, Michael .

BRITISH JOURNAL OF HAEMATOLOGY, 2011, 154 (02) :208-215

[8] Current concepts in the prevention of pathogen transmission via blood/plasma-derived products for bleeding disorders [J].

Di Minno, Giovanni ;

Perno, Carlo Federico ;

Tiede, Andreas ;

Navarro, David ;

Canaro, Mariana ;

Gueertler, Lutz ;

Ironside, James W. .

BLOOD REVIEWS, 2016, 30 (01) :35-48

[9] Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort [J].

Feldman, Brian M. ;

Rivard, Georges E. ;

Babyn, Paul ;

Wu, John K. M. ;

Steele, MacGregor ;

Poon, Man-Chiu ;

Card, Robert T. ;

Israels, Sara J. ;

Laferriere, Nicole ;

Gill, Kulwant ;

Chan, Anthony K. ;

Carcao, Manuel ;

Klaassen, Robert J. ;

Cloutier, Stephanie ;

Price, Victoria E. ;

Dover, Saunya ;

Blanchette, Victor S. .

LANCET HAEMATOLOGY, 2018, 5 (06) :E252-E260

[10] Prospective observational cohort studies for studying rare diseases: the European PedNet Haemophilia Registry [J].

Fischer, K. ;

Ljung, R. ;

Platokouki, H. ;

Liesner, R. ;

Claeyssens, S. ;

Smink, E. ;

Van den Berg, H. M. .

HAEMOPHILIA, 2014, 20 (04) :e280-e286

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