A型血友病是一种遗传性血凝症，患者缺少一种可产生凝血因子VIII的基因。患者可能会发生频繁的严重出血，多发于关节，有可能导致关节严重受损。
  2018年2月27日，瑞士罗氏集团表示，欧盟委员会已批准其药物Hemlibra(Emicizumab)用于对标准治疗产生了抗药性的血友病患者。今年1月，该药物获得欧盟专家组推荐。去年该药已经在美国获得批准。
  关于Emicizumab：
  Emicizumab是一种双特异性单克隆抗体，旨在将激活天然凝血级联所需的蛋白质聚集在一起，并恢复血友病A患者的凝血过程。
  Emicizumab可用于预防或减少已经发展出称为因子VIII（FVIII）抑制剂的成年和儿童A型血友病患者出血事件的频率。

Hemlibra (Emicizumab-Kxwh Injection)
HEMLIBRA Rx
Generic Name and Formulations:
Emicizumab-kxwh 30mg/mL, 60mg/0.4mL, 105mg/0.7mL, 150mg/mL; per vial; soln for SC inj; preservative-free.
Company:
Genentech, Inc.
Indications for HEMLIBRA:
Routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A with factor VIII inhibitors.
Adults and Children:
Do not inj into moles, scars, tender skin, bruised, red, hard or not intact skin areas. Give by SC inj into upper outer arms, thighs, or any abdomen quadrant; rotate inj sites. 3mg/kg once weekly for first 4 weeks, then 1.5mg/kg once weekly.
Warnings/Precautions:
Thrombotic microangiopathy and thromboembolism can occur when average cumulative of >100U/kg per 24hrs of activated prothrombin complex concentrate (aPCC) was given for ≥24hrs. Females of reproductive potential should use effective contraception during treatment. Pregnancy. Nursing mothers.
Interactions:
Risk of thrombotic microangiopathy and thromboembolism with concomitant aPCC; monitor and immediately discontinue if occurs. May interfere with coagulation lab tests (eg, ACT, aPTT, aPTT-based assays, Bethesda assays [clotting-based] for FVIII inhibitor titers). Possible hypercoagulability with concomitant recombinant FVIIa or FVIII.
Pharmacological Class:
Bispecific factor IXa- and factor X-directed antibody.
Adverse Reactions:
Inj site reactions, headache, arthralgia, pyrexia, diarrhea, myalgia; thrombotic microangiopathy, thromboembolism.
Generic Availability:
NO
How Supplied:
Single-dose vial—1