[Advances in hemophilia treatment].

Keiji Nogami
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Abstract

Advances in replacement therapy with clotting factor (F) VIII or FIX product have contributed greatly to reducing the incidence of hemophilic arthropathy and improving quality of life (QOL) in patients with hemophilia. However, frequent intravenous administration of clotting factor products, blood access, and development of alloantibodies (inhibitors) have been important issues. Clinical studies aimed at addressing these issues have been conducted in Japan as well, including a multicenter study to determine factors involved in inhibitor development. Drug development has also progressed: several clotting factor products with extended half-life and non-clotting factor therapies have been introduced in quick succession. Anti-FIX/FX bispecific antibody in particular has a long half-life when administered subcutaneously and controls bleeding in patients with hemophilia A. Anti-antithrombin therapy and anti-TFPI monoclonal antibody products that work by rebalancing coagulation have also been developed. In addition, gene therapy has been approved for adults in U.S. and Europe, where improved vectors and codon optimization have enabled protein expression up to the near-therapeutic hemostatic range. Recent significant developments in hemophilia treatment are expected to overcome long-standing problems and further improve QOL.

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[血友病治疗进展]。
凝血因子 (F) VIII 或 FIX 产品替代疗法的进步极大地降低了血友病关节病的发病率,改善了血友病患者的生活质量(QOL)。然而,频繁静脉注射凝血因子产品、血液获取和异体抗体(抑制剂)的产生一直是个重要问题。日本也开展了旨在解决这些问题的临床研究,包括一项确定抑制剂产生因素的多中心研究。药物开发也取得了进展:几种延长半衰期的凝血因子产品和非凝血因子疗法相继问世。抗 FIX/FX 双特异性抗体的皮下注射半衰期较长,可控制 A 型血友病患者的出血,抗抗凝血酶疗法和抗 TFPI 单克隆抗体产品也已开发出来,它们通过重新平衡凝血发挥作用。此外,基因疗法已在美国和欧洲获准用于成人患者,通过改进载体和优化密码子,使蛋白质表达量接近治疗止血范围。血友病治疗领域的最新重大进展有望克服长期存在的问题,进一步改善患者的生活质量。
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