Immune thrombocytopenia (ITP) is a common autoimmune bleeding disorder characterized by accelerated platelet destruction and impaired platelet production due to antibody-mediated mechanisms. Current first-line therapies for ITP include glucocorticoids and immunoglobulins, while second-line options often involve thrombopoietin receptor agonists (TPO-RAs), CD20 monoclonal antibodies, and splenectomy. The introduction of CD20 monoclonal antibody therapy over the past decade has transformed ITP treatment. However, heterogeneity in treatment response and duration leaves a subset of patients at risk of severe bleeding, reduced quality of life, and increased mortality. Following the success of CD20 antibodies, researchers have been exploring other potent therapeutic targets to address these unmet needs.
CD38-positive long-lived plasma cells (LLPCs) play a critical role in ITP by continuously producing pathogenic antibodies, leading to disease relapse or refractory cases. Targeting CD38 to eliminate LLPCs and other antibody-secreting cells offers a promising therapeutic strategy for achieving durable clinical benefits in ITP.
On June 19, 2024, Professors Lei Zhang and Renchi Yang’s team from the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, published a groundbreaking study in The New England Journal of Medicine (Impact Factor: 158.5) titled “A Novel Anti-CD38 Monoclonal Antibody for Treating Immune Thrombocytopenia.” This study represents the first global report evaluating the efficacy, safety, and mechanism of CM313, a novel CD38-targeting monoclonal antibody for ITP treatment. The study marks a major innovation in ITP research and has the potential to reshape international treatment guidelines.
