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摘要

目前对A型血友病患者的治疗比以往更安全、更有效。预防性替代涉及反复静脉给药血浆源性因子VIII或重组因子VIII产品,带来不便和可能的不良反应。抑制剂的出现需要激活凝血酶原复合物浓缩物或激活因子VII -一种昂贵的治疗方法。免疫耐受诱导是高滴度抑制剂患者的理想治疗方法,是消除抑制剂的唯一潜在方法,但非常昂贵。由于这些原因,医学界对科学研究在新分子领域所取得的进展很感兴趣,因为新分子不像目前的替代疗法那样带来不便,而且未来可能会取代它。本文的目的是简要回顾新的治疗可能性的血友病A,可以预防潜在的关节外出血,避免抑制剂的发生,并有尽可能少的不良反应。
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A Look at the Future Hemophilia A Treatment
The current treatment of patients with hemophilia A is safer and more effective than the previous one. Prophylactic substitution involves repeated intravenous administration of plasma-derived factor VIII or recombinant factor VIII products, with inconveniences and possible adverse effects. The occurrence of inhibitors requires the administration of activated prothrombin complex concentrate or activated factor VII - an expensive treatment. The immune tolerance induction is the ideal treatment for patients with high titres of inhibitors - the only potential way to eliminate inhibitors and very expensive. For these reasons, the medical world is interested in the advances that scientific research is doing in the field of new molecules without the inconveniences of current substitution therapy and which could replace it in the future. The purpose of this article is to briefly review the new therapeutic possibilities for patients with hemophilia A, which can prevent potential extraarticular bleedings, avoid the occurrence of inhibitors, and have as few adverse effects as possible.
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