CD19 CAR-T therapy induces remission in refractory autoimmune hemolytic anemia with ITP and antiphospholipid syndrome

Korte I, Kharboutli S, Völkl S, Heberling L, Scholz JK, Pfeiffer H, Strobel J, Harrer T, Kretschmann S, Vasova I, Gerbitz A, Achenbach S, Spriewald B, Krause S, Gutsche K, von Bonin M, Röllig C, Aigner M, Mackensen A, Müller F (2026)

Publication Type: Journal article

Publication year: 2026

Journal

Med Cell Press

Article Number: 101075

DOI: 10.1016/j.medj.2026.101075

Abstract

Background: Autoimmune hemolytic anemia (AIHA), immune thrombocytopenia (ITP), and antiphospholipid antibody syndrome (APLAS) are B cell-driven autoimmune diseases defined by pathogenic autoantibodies. CD19-directed chimeric antigen receptor (CAR)-T cells have recently demonstrated the ability to reset dysregulated B cells and induce long-lasting remission in refractory systemic autoimmune diseases. Evidence for efficacy in severe, treatment-refractory AIHA, however, is limited. Methods: We report the clinical course of a 47-year-old woman with life-threatening cold- and warm-agglutinin AIHA refractory to nine prior treatment lines, accompanied by ITP and APLAS. In an uncontrolled flare, she received a fludarabine/cyclophosphamide-containing lymphodepletion followed by autologous CD19-directed, 4-1BB-costimulated CAR-T cells (zorpocabtagene-autoleucel [Zorpo-cel], 1 × 106/kg) on the basis of compassionate use. Treatment efficacy and safety were assessed over an 11-month follow-up period. Findings: Zorpo-cel showed a rapid and sustained B cell depletion. Transfusion independence was achieved by day 7, with hemoglobin normalization by day 25, including resolution of hemolysis markers. Cold-agglutinin titers decreased, and previously elevated antiphospholipid antibodies normalized without recurrence throughout 11 months of follow-up. ITP stabilized. No cytokine release syndrome or neurotoxicity occurred. Mild transaminase elevation and thrombocytopenia were observed, most likely correlating with pre-existing severe iron overload due to erythrocyte transfusions. Conclusion: This case demonstrates that CD19-directed CAR-T cell therapy can induce rapid, durable remission of severe, refractory cold-agglutinin AIHA and simultaneously improve coexisting APLAS and ITP on a favorable toxicity profile. However, more data from controlled clinical trials are needed for final conclusions. Funding: This work was funded by Deutsche Forschungsgemeinschaft and Deutsche Krebshilfe.

Authors with CRIS profile

Isabel Korte Department of Medicine 5 – Haematology and Oncology Simon Völkl Department of Medicine 5 – Haematology and Oncology Julia Scholz Department of Medicine 5 – Haematology and Oncology Hella Pfeiffer Department of Transfusion Medicine and Haemostaseology Julian Strobel Department of Transfusion Medicine and Haemostaseology Thomas Harrer Department of Medicine 3 – Rheumatology and Immunology Sascha Kretschmann Department of Medicine 5 – Haematology and Oncology Ingrid Vasova Lehrstuhl für Innere Medizin V Armin Gerbitz Department of Medicine 5 – Haematology and Oncology Susanne Achenbach Department of Transfusion Medicine and Haemostaseology Bernd Spriewald Department of Medicine 5 – Haematology and Oncology Stefan Krause Department of Medicine 5 – Haematology and Oncology Andreas Mackensen Lehrstuhl für Innere Medizin V Fabian Müller Department of Medicine 5 – Haematology and Oncology

Involved external institutions

Universitätsklinikum Carl Gustav Carus Dresden DE Germany (DE)

How to cite

APA:

Korte, I., Kharboutli, S., Völkl, S., Heberling, L., Scholz, J.K., Pfeiffer, H.,... Müller, F. (2026). CD19 CAR-T therapy induces remission in refractory autoimmune hemolytic anemia with ITP and antiphospholipid syndrome. Med. https://doi.org/10.1016/j.medj.2026.101075

MLA:

Korte, Isabel, et al. "CD19 CAR-T therapy induces remission in refractory autoimmune hemolytic anemia with ITP and antiphospholipid syndrome." Med (2026).

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