CD19 CAR-T 细胞治疗小鼠免疫性血小板减少症

Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by antiplatelet autoantibodies, with many patients refractory or relapsing on conventional treatments. GPIb , an important autoantigen in ITP, is notably linked to refractoriness, highlighting the need for novel treatments.

We assessed CD19 chimeric antigen receptor (CAR)-T cell therapy's potential in a modified murine model targeting GPIb . CD19 CAR-T cell infusion accelerated platelet count recovery compared to the control group, effectively depleted CD19 + B cells and CD138 + plasma cells, and markedly reduced anti-GPIb autoantibodies in vivo. In vitro CD19 CAR-T cells reduced both plasma cells and B cells in the spleens of mice and ITP patients.

CD19 CAR-T cell therapy significantly altered T-cell subsets, increasing regulatory T cells, T helper 1 and T helper 17 populations, suggesting a role in modulating the immune response for sustained ITP remission. Monitoring of body/spleen weights and temperature showed no significant cytokine release syndrome, indicating a favourable safety profile. These promising results support the potential of CD19 CAR-T cell therapy as a novel treatment option for refractory ITP, particularly in GPIb -positive autoantibody patients.

Further clinical studies are warranted to assess the safety and efficacy of this approach in human patients.