Factor VIII replacement is still the standard of care in haemophilia A

被引：95

作者：

Aledort, Louis ^{[1
]}

Mannucci, Pier Mannuccio ^{[2
]}

Schramm, Wolfgang ^{[3
]}

Tarantino, Michael ^{[4
,5
]}

机构：

[1] Icahn Sch Med Mt Sinai, Div Hematol Oncol, New York, NY 10029 USA

[2] IRCCS Fdn Ca Granda Maggiore Policlin Hosp, Sci Direct, Via Pace 9, I-20122 Milan, Italy

[3] Univ Hosp Munich, Dept Transfus Med & Haemostasis, Munich, Germany

[4] Univ Illinois, Coll Med, Peoria, IL 61656 USA

[5] Bleeding & Clotting Disorders Inst, Peoria, IL USA

来源：

BLOOD TRANSFUSION | 2019年 / 17卷 / 06期

关键词：

factor VIII; haemophilia A; prophylaxis; standard of care; half-life; RECOMBINANT FACTOR-VIII; FC FUSION PROTEIN; EXTENDED-HALF-LIFE; BAY; 94-9027; EMICIZUMAB PROPHYLAXIS; BISPECIFIC ANTIBODY; IMMUNE TOLERANCE; FULL-LENGTH; ON-DEMAND; SAFETY;

D O I：

10.2450/2019.0211-19

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Prophylactic factor VIII (FVIII) has dramatically improved haemophilia A treatment, preventing joint bleeding and halting the deterioration of joint status. FVIII products with an extended plasma half-life further improve patients' quality of life and increase therapeutic adherence. New licensed classes of non-replacement products include prophylactic emicizumab, which is administered subcutaneously up to every 4 weeks. However, this drug is not suitable for acute bleeding episodes or management of major surgery, and long-term data on the impact of emicizumab on joint health, FVIII inhibitor development and thrombotic risk are awaited. Prophylaxis with FVIII replacement remains the standard of care in haemophilia A, with the aim of achieving a level of haemostasis control that allows patients to meet their lifestyle goals.

引用

页码：479 / 486

页数：8

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