2026年中国首篇NEJM论文：CAR-T细胞治疗自身免疫溶血性贫血

Core Viewpoint - The study demonstrates that CD19-targeted CAR-T cell therapy shows promising efficacy in treating refractory autoimmune hemolytic anemia (AIHA), with all 11 patients achieving complete response, marking a significant breakthrough in this field [3][6]. Group 1: Study Overview - The research involved 11 patients with refractory AIHA who had previously undergone at least three lines of treatment without success [6]. - The therapy utilized autologous CD19 CAR-T cells developed by Healion Biotech, which has been approved for treating relapsed/refractory B-cell acute lymphoblastic leukemia and large B-cell lymphoma [6]. - The primary aim of the study was to assess safety characteristics, including the incidence and severity of adverse events such as cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome [6]. Group 2: Efficacy Results - All 11 patients achieved complete response, with a median time to complete response of 45 days (range 21-153 days) [7]. - The median duration of drug-free remission was 11.5 months (range 6.8-21.0 months) [7]. - The study provides the first global evidence of the efficacy of CD19 CAR-T cell therapy in the AIHA domain, offering new hope for patients with recurrent or refractory AIHA [3]. Group 3: Safety Profile - Nine patients experienced grade 1-2 cytokine release syndrome, and one patient had grade 1 immune effector cell-associated neurotoxicity syndrome [9]. - A total of 15 infection events occurred among seven patients, with no grade 4 or higher infections reported [9]. - The study identified a specific B cell microenvironment associated with relapse, characterized by initial B cells and interactions with HLA-DRB5+ B cells and CD4+ T cells [9]. Group 4: Conclusion - Overall, the clinical study indicates that CD19 CAR-T cell therapy produces expected toxic effects while providing sustained remission in patients with multi-drug refractory AIHA [11].