替克司他单抗治疗抗 CD19 CAR-T 细胞疗法失败后复发难治性获得性血友病 A
Teclistamab in relapsed, multi-refractory acquired hemophilia A after failure of anti-CD19 CAR-T cell therapy
获得性血友病 A(AHA)是一种自身免疫性出血病,患者体内产生针对凝血因子的抑制物。本文报告一位复发患者,此前接受过多种免疫抑制治疗,包括自体抗 CD19 嵌合抗原受体(CAR)T 细胞疗法,均告失败。患者随后接受替克司他单抗——一种双特异性单克隆抗体,可把自体 CD3 阳性 T 细胞与浆细胞及成熟 B 细胞表面的 B 细胞成熟抗原(BCMA)连接起来。治疗耐受良好,除低丙种球蛋白血症外无临床显著不良事件,患者获得迅速且持久的缓解,持续一年以上。这是替克司他单抗成功治疗难治性 AHA 的首个病例,为靶向浆细胞的双特异性抗体提供了概念验证。
为什么推荐给您:首次用双特异性抗体成功治疗 CAR-T 失败的难治性血友病,属新机制概念验证。
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摘要Abstract
A patient with relapsed acquired hemophilia A (AHA), who previously failed multiple lines of immunosuppressive therapy (IST) including autologous anti-CD19 chimeric antigen receptor (CAR) T cell therapy received teclistamab, a bispecific monoclonal antibody engaging autologous CD3-positive T cells with the B cell maturation antigen (BCMA) on plasma cells and mature B cells. Teclistamab was well tolerated without clinically significant adverse events, except for hypogammaglobulinemia. Prompt and durable remission was observed that lasted for more than one year post treatment. This is the first case of refractory AHA successfully treated with teclistamab, providing a proof-of-concept of plasma cell-directed bispecific antibodies, supporting its further evaluation.