Nothing short of a revolution: Novel extended half-life factor VIII replacement products and non-replacement agents reshape the treatment landscape in hemophilia A
Hussien Ahmed H. Abdelgawad , Rachel Foster , Mario Otto
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引用次数: 0
Abstract
Hemophilia A, an X-linked genetic disorder, is characterized by a deficiency or dysfunction of clotting Factor VIII. The treatment landscape has substantially changed by introducing novel extended half-life factor VIII (EHL-FVIII) replacement therapies such as efanesoctocog Alfa and non-factor replacement therapy such as emicizumab. These agents signal a shift from treatments requiring multiple weekly infusions to advanced therapies with long half-lives, offering superior protection against bleeding and improving patient adherence and quality of life. While EHL-FVIII treatment might lead to inhibitor development in some patients, non-factor replacement therapy carries thrombotic risks. Therefore, ongoing research and the generation of robust clinical evidence remain vital to guide the selection of optimal and cost-effective first-line therapies for hemophilia A patients.
血友病 A 是一种 X 连锁遗传疾病,其特征是凝血因子 VIII 缺乏或功能障碍。通过引入新型延长半衰期因子 VIII(EHL-FVIII)替代疗法(如 efanesoctocog Alfa)和非因子替代疗法(如 emicizumab),治疗格局发生了重大变化。这些药物标志着从需要每周多次输注的治疗方法转变为具有长半衰期的先进疗法,从而提供更好的出血保护,并提高患者的依从性和生活质量。虽然 EHL-FVIII 治疗可能会导致某些患者出现抑制剂,但非因子替代疗法也存在血栓风险。因此,持续的研究和可靠的临床证据对于指导 A 型血友病患者选择最佳且经济有效的一线疗法仍然至关重要。
期刊介绍:
Blood Reviews, a highly regarded international journal, serves as a vital information hub, offering comprehensive evaluations of clinical practices and research insights from esteemed experts. Specially commissioned, peer-reviewed articles authored by leading researchers and practitioners ensure extensive global coverage across all sub-specialties of hematology.