Department of Hematology and Transfusion Medicine, C. Poma Hospital, Mantova, Italy.
Semin Thromb Hemost. 2013 Oct;39(7):772-8. doi: 10.1055/s-0033-1354425. Epub 2013 Sep 8.
The management of patients with congenital hemophilia who develop alloantibodies that neutralize coagulation factor activity is the most important challenge for hemophilia care providers because this complication renders replacement treatment with factor concentrates partially or completely ineffective, exposing the patients to an increased risk of morbidity and mortality. Development of inhibitors complicates the clinical course of severe hemophilia in up to 30% of patients with hemophilia A and up to 5% of those with hemophilia B. Although the ultimate goal of treatment of patients with alloantibodies against factors VIII and IX is eradication of the inhibitor, the control of bleeding through high doses of factor concentrates (low titer inhibitors) or bypassing agents (high titer inhibitors) is the mainstay of management of these patients. In this review, we summarize the main characteristics of the bypassing agents FEIBA and NovoSeven, briefly discussing available literature data, and in particular, focusing on comparative studies.
患有先天性血友病并产生中和凝血因子活性的抗体的患者的管理是血友病护理提供者面临的最大挑战,因为这种并发症使因子浓缩物的替代治疗部分或完全无效,使患者面临更高的发病率和死亡率风险。抑制剂的发展使多达 30%的甲型血友病患者和多达 5%的乙型血友病患者的严重血友病的临床病程复杂化。尽管针对因子 VIII 和因子 IX 的抗体的患者的治疗的最终目标是消除抑制剂,但通过高剂量的因子浓缩物(低滴度抑制剂)或旁路剂(高滴度抑制剂)控制出血是这些患者管理的主要方法。在这篇综述中,我们总结了旁路剂 FEIBA 和诺维赞的主要特征,简要讨论了现有文献数据,特别是侧重于比较研究。