Salvage treatment of multi-refractory primary immune thrombocytopenia with CD19 CAR T cells
Primary immune thrombocytopenia is an autoimmune disease in which autoreactive B cells play a crucial role in pathogenesis by producing autoantibodies primarily directed against platelet surface glycoproteins (eg, glycoprotein IIb/IIIa; fibrinogen receptor) that trigger platelet destruction, resulting in severe thrombocytopenia. The annualised incidence of primary immune thrombocytopenia is approximately 3·3 cases per 100 000 people among adults.1 Despite several treatment options, including approved thrombopoietin receptor agonists and commonly used antibody-reducing approaches—such as B-cell depletion with the anti-CD20 monoclonal antibody rituximab, BTK inhibitors, SYK inhibitors, corticosteroids, and splenectomy—a subset of patients develop refractory immune thrombocytopenia.
