Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt, Germany.
Department of Haemostaseology and Haemophilia Center, Internal Medicine, Frankfurt, Germany.
Expert Opin Biol Ther. 2021 Nov;21(11):1395-1402. doi: 10.1080/14712598.2021.1908993. Epub 2021 Apr 1.
: The standard treatment of hemophilia A consists of the prophylactic administration of a coagulation factor concentrate, to be administered intravenously several times a week. Newly approved factor concentrates and non-factor products reduce the frequency of injection and offer better protection against bleeding.: New treatment options for hemophilia A are either coagulation factor concentrates based on innovative active principles extending half-life (EHL) or non-factor products allowing subcutaneous application with an extended half-life, so that their broader application only needs to be made every one to four weeks. Other new therapeutic options are still in clinical studies, such as the inhibition of TFPI (tissue factor pathway inhibitor) or small interfering mRNA molecule against antithrombin and gene therapy for hemophilia A.: It can be expected that patients with hemophilia will benefit significantly from the new treatment options and that the protection against bleeding and joint damage as well as the quality of life will increase. The availability of alternatives to classical replacement therapy will require the development of treatment algorithms for patients with hemophilia. It is still unclear to what extent factor substitution will be challenged by the new therapies as first-line therapy.
: 血友病 A 的标准治疗包括预防性输注凝血因子浓缩物，每周需静脉注射数次。新批准的因子浓缩物和非因子产品减少了注射频率，并提供了更好的出血预防效果。: 血友病 A 的新治疗选择是基于延长半衰期（EHL）的创新活性原理的凝血因子浓缩物，或允许皮下应用延长半衰期的非因子产品，因此它们的更广泛应用只需每一到四周进行一次。其他新的治疗选择仍处于临床研究阶段，例如组织因子途径抑制剂（TFPI）的抑制或针对抗凝血酶的小干扰 mRNA 分子和血友病 A 的基因治疗。: 可以预期，新的治疗选择将使血友病患者显著受益，并且对出血和关节损伤的保护以及生活质量将得到提高。替代经典替代疗法的选择将需要为血友病患者制定治疗算法。新疗法作为一线治疗在多大程度上会挑战因子替代仍不清楚。
