Mount Sinai School of Medicine, One Gustave L. Levy Place, Box 1006, Newyork, NY, USA.
Expert Rev Hematol. 2014 Jun;7(3):373-85. doi: 10.1586/17474086.2014.899896. Epub 2014 Apr 10.
Hemophilia A is a congenital, recessive, X-linked bleeding disorder that is managed with infusions of plasma-derived or recombinant factor (F) VIII. The primary considerations in FVIII replacement therapy today are the: 1) immunogenicity of FVIII concentrates, 2) role of longer-acting FVIII products, 3) prophylactic use of FVIII in children and adults with severe hemophilia A, and 4) affordability and availability of FVIII products. Improving patient outcomes by increasing the use of FVIII prophylaxis, preventing or eliminating FVIII inhibitors, and expanding access to FVIII concentrates in developing countries are the major challenges confronting clinicians who care for patients with hemophilia A.
血友病 A 是一种先天性、隐性、X 连锁的出血性疾病，通过输注血浆衍生或重组因子 (F)VIII 进行治疗。目前，FVIII 替代治疗的主要考虑因素包括：1）FVIII 浓缩物的免疫原性，2）长效 FVIII 产品的作用，3）在患有严重血友病 A 的儿童和成人中预防性使用 FVIII，以及 4）FVIII 产品的可负担性和可得性。通过增加 FVIII 预防的使用、预防或消除 FVIII 抑制剂以及扩大发展中国家 FVIII 浓缩物的可及性，改善患者的预后，是治疗血友病 A 患者的临床医生面临的主要挑战。
