Division of Hematology/Oncology, The Hospital for Sick Children, Department of Pediatrics, University of Toronto, Toronto, ON, Canada.
Haemophilia. 2010 May;16 Suppl 3:46-51. doi: 10.1111/j.1365-2516.2010.02260.x.
For patients with haemophilia, the development of inhibitors complicates treatment, and inhibitor patients may thus have a range of unmet needs. Although successful inhibitor eradication will render patients responsive to factor replacement therapy, with potentially beneficial effects on long-term outcomes, this may not always be possible. Physicians treating inhibitor patients should aim to achieve reliable control of bleeding episodes, and the prevention of joint disease should also be a priority. Patients with high-titre inhibitors require therapy with bypassing agents--recombinant activated factor VII (rFVIIa) or a plasma-derived activated prothrombin complex concentrate (pd-APCC)--for the treatment of bleeding. When treating joint haemorrhage in inhibitor patients, both aggressive treatment of intercurrent joint bleeds and prophylaxis should be considered, although evidence is needed as to whether prophylaxis with bypassing agents can significantly delay/prevent the development of osteochondral changes in patients with inhibitors. Despite physicians' best efforts, joint disease may ultimately occur in inhibitor patients, and in such instances optimizing treatment, of both early and late stages, is important. There is no single therapeutic modality for dealing with the various treatment challenges posed by inhibitor patients, but overall goals should be to improve quality of life, with the provision of cost-effective care that aims to maintain physical function.
对于血友病患者来说，抑制剂的产生会使治疗复杂化，因此这些患者可能存在一系列未满足的需求。虽然成功消除抑制剂会使患者对因子替代疗法产生反应，从而对长期预后产生潜在的有益影响，但这并不总是可行的。治疗抑制剂患者的医生应旨在实现出血事件的可靠控制，预防关节疾病也应是优先事项。高滴度抑制剂患者需要使用旁路制剂（重组活化因子 VII（rFVIIa）或血浆衍生的活化凝血酶原复合物浓缩物（pd-APCC））治疗出血。在治疗抑制剂患者的关节出血时，应考虑积极治疗关节内出血和预防措施，尽管需要有证据表明旁路制剂的预防是否能显著延缓/预防抑制剂患者的骨软骨变化。尽管医生尽了最大努力，关节疾病最终可能会发生在抑制剂患者身上，在这种情况下，优化早期和晚期的治疗非常重要。对于抑制剂患者带来的各种治疗挑战，没有单一的治疗方法，但总体目标应该是提高生活质量，提供具有成本效益的护理，旨在维持身体功能。
