[Chinese guidance for the clinical application of Adeno-associated virus vector-based gene therapy for hemophilia B (2025)].

Abstract

The mainstay of treatment for hemophilia B is Factor Ⅸ or non-factor drug therapy; however, as the disease is incurable, lifelong treatment is required. In recent years, gene therapy for hemophilia B has advanced significantly, with three adeno-associated virus (AAV) vector-based gene therapy products receiving market authorization globally. Among these, Palbociclib was recently approved in China. AAV vector gene therapy is characterized by its irreversible post-treatment effects and potential for long-term efficacy; however, suboptimal efficacy or loss of efficacy has been observed in some cases during early clinical trials. Eligibility for AAV vector gene therapy primarily depends on several factors, including patient diagnostic subtype, age, inhibitor status, AAV capsid antibody titer, and the preferences of the patient and/or their family. Given that AAV vector-based gene therapy for hemophilia has become an accessible frontier treatment, the Thrombosis and Hemostasis Group of the Chinese Society of Hematology, Chinese Medical Association, and the Hemophilia Treatment Center Collaborative Network of China have jointly formulated this guidance. This guidance aims to standardize operational procedures and follow-up recommendations, ensuring that patients receive standardized management when undergoing this novel therapeutic approach.