Kempton Christine L, White Gilbert C
AFLAC Cancer Center and Blood Disorders Service and Winship Cancer Institute, Emory University, Atlanta, GA 30322, USA.
Blood. 2009 Jan 1;113(1):11-7. doi: 10.1182/blood-2008-06-160432. Epub 2008 Sep 26.
The most significant complication of treatment in patients with hemophilia A is the development of alloantibodies that inhibit factor VIII activity. In the presence of inhibitory antibodies, replacement of the missing clotting factor by infusion of factor VIII becomes less effective. Once replacement therapy is ineffective, acute management of bleeding requires agents that bypass factor VIII activity. Long-term management consists of eradicating the inhibitor through immune tolerance. Despite success in the treatment of acute bleeding and inhibitor eradication, there remains an inability to predict or prevent inhibitor formation. Ideally, prediction and ultimately prevention will come with an improved understanding of how patient-specific and treatment-related factors work together to influence anti-factor VIII antibody production.
甲型血友病患者治疗最严重的并发症是产生抑制凝血因子 VIII 活性的同种抗体。在存在抑制性抗体的情况下,通过输注凝血因子 VIII 替代缺失的凝血因子效果会变差。一旦替代疗法无效,急性出血的处理需要使用能绕过凝血因子 VIII 活性的药物。长期管理包括通过免疫耐受消除抑制剂。尽管在急性出血治疗和消除抑制剂方面取得了成功,但仍无法预测或预防抑制剂的形成。理想情况下,随着对患者特异性因素和治疗相关因素如何共同作用影响抗凝血因子 VIII 抗体产生的理解不断加深,将能够实现预测并最终预防。