Zhang L
National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
Zhonghua Xue Ye Xue Za Zhi. 2025 May 14;46(5):385-388. doi: 10.3760/cma.j.cn121090-20250304-00111.
Hemophilia B is a monogenic inherited disorder characterized by spontaneous or trauma-induced bleeding. In China, the primary treatment strategy for hemophilia B is factor Ⅸ (FⅨ) replacement therapy, requiring patients to undergo frequent venipunctures throughout their lifetime. In recent years, significant advancements have been made in gene therapy for hemophilia B. As gene therapy for hemophilia B is nearing clinical implementation in China, the Thrombosis and Hemostasis Group of the Chinese Society of Hematology of the Chinese Medical Association and the Hemophilia Treatment Center Collaborative Network of China have jointly developed the "Chinese guidance for clinical application of Adeno-associated virus vector-based gene therapy for hemophilia B (2025)", offering comprehensive recommendations for standardizing the entire gene therapy process. This article will introduce the background, key points, and clinical value of the guidelines to enhance awareness and emphasis on gene therapy for hemophilia B in China.
血友病B是一种单基因遗传性疾病，其特征为自发性出血或创伤后出血。在中国，血友病B的主要治疗策略是凝血因子Ⅸ（FⅨ）替代疗法，这要求患者终生频繁接受静脉穿刺。近年来，血友病B的基因治疗取得了重大进展。随着血友病B的基因治疗在中国即将进入临床应用阶段，中华医学会血液学分会血栓与止血学组和中国血友病治疗中心协作网络联合制定了《腺相关病毒载体介导的血友病B基因治疗临床应用中国指导原则（2025年版）》，为规范整个基因治疗过程提供了全面建议。本文将介绍该指南的背景、要点及临床价值，以提高中国对血友病B基因治疗的认识和重视程度。
