Autologous CD19 CAR T cell treatment of multirefractory AIHA
Li R, Pan H, Zhang L, et al. CD19 CAR T-Cell Therapy for Autoimmune Hemolytic Anemia. New England Journal of Medicine. 2026; (doi: 10.1056/NEJMoa2509820).
New study results demonstrate CD19 chimeric antigen receptor (CAR) T-cell therapy as a promising therapeutic pathway for autoimmune hemolytic anemia (AIHA), which is notoriously prone to relapse. Advanced, multirefractory cases are recognized by failed response to three or more lines of treatment against persistent autoreactive B-cell activity, but investigators believe CAR T-cell therapy, which achieves significant B-cell depletion, represents a viable drug-free alternative in this population. All 11 trial participants (five from a compassionate-use program and six from a Phase I study) underwent a single infusion of autologous CD19 CAR T cells, and each recorded a complete response. Over a median follow-up of 12.2 months, the median duration of drug-free remission was 11.5 months, and toxic effects were in line with expectations. Cytokine release syndrome developed in nine patients, at grades 1 or 2; one patient presented with immune effector cell-associated hematotoxicity, grade 3; and seven patients developed a total of 15 infections, all categorized as grade 3 or lower.