Turoctocog alfa治疗A型血友病患者:III期试验收集的生活质量数据回顾

S. Seremetis, R. Kulkarni, A. Regnault, E. Santagostino
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引用次数: 1

摘要

血友病A是一种由凝血因子VIII缺乏引起的x连锁隐性遗传性出血性疾病。血友病及其治疗的困难对患者的生活质量提出了挑战。Turoctocog alfa是一种重组的b结构域截断因子VIII,是最近获得美国FDA和ema批准的替代疗法,被证明是治疗a型血友病个体的有效和安全的选择。尤其是年轻人,当从按需治疗转为预防性治疗时,他们的健康相关生活质量得到了改善。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Turoctocog alfa in the treatment of individuals with hemophilia A: review of quality of life data collected in Phase III trials
Hemophilia A is an X-linked recessive hereditary bleeding disorder resulting from a deficiency in coagulation factor VIII. Difficulties due to hemophilia and its management present challenges for patient's quality of life. Turoctocog alfa, a recombinant, B-domain truncated factor VIII, is a recent US FDA- and EMA-approved replacement therapy shown to be an effective and safe option for the treatment of individuals with hemophilia A. Data collected throughout two Phase 3, multinational, open-label, non-randomized, non-comparative trials demonstrated that individuals with hemophilia A, particularly young adults experienced improvements in health-related quality of life when switched from an on-demand to a prophylactic regimen of turoctocog alfa.
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