Shapiro Amy D
1Indiana Hemophilia and Thrombosis Center, Indianapolis, IN; and.
Hematology Am Soc Hematol Educ Program. 2013;2013:37-43. doi: 10.1182/asheducation-2013.1.37.
In the past 50 years, the lifespan of an individual affected with severe hemophilia A has increased from a mere 20 years to near that of the general unaffected population. These advances are the result of and parallel advances in the development and manufacture of replacement therapies. We are now poised to witness further technologic leaps with the development of longer-lasting replacement therapies, some of which are likely to be approved for market shortly. Prophylactic therapy is currently the standard of care for young children with severe hemophilia A, yet requires frequent infusion to achieve optimal results. Longer-lasting products will transform our ability to deliver prophylaxis, especially in very young children. Longer-lasting replacement therapies will require changes to our current treatment plans including those for acute bleeding, prophylaxis, surgical interventions, and even perhaps immunotolerance induction. Ongoing observation will be required to determine the full clinical impact of this new class of products.
在过去50年里，重度甲型血友病患者的寿命已从仅仅20岁延长至接近普通未患病人群的寿命。这些进步得益于替代疗法研发和生产方面的进步，并且与之并行发展。随着更长效替代疗法的开发，我们即将见证进一步的技术飞跃，其中一些疗法可能很快会获批上市。预防性治疗目前是重度甲型血友病幼儿的标准治疗方法，但需要频繁输注才能取得最佳效果。更长效的产品将改变我们提供预防性治疗的能力，尤其是对非常年幼的儿童。更长效的替代疗法将需要改变我们目前的治疗方案，包括急性出血、预防、手术干预，甚至可能还有免疫耐受诱导方面的方案。需要持续观察以确定这类新产品的全面临床影响。
