Daratumumab in patients with immune thrombocytopenia: a single-center, open-label, phase 2 trial.

Background: Immune thrombocytopenia is an autoimmune bleeding disorder. Daratumumab, a human monoclonal antibody targeting CD38 glycoprotein, is currently under clinical investigation as a potential therapy for autoimmune diseases. This trial aimed to evaluate the efficacy and safety of daratumumab in adults with immune thrombocytopenia. Methods: This single-center, open-label, phase 2 trial enrolled patients with a platelet count <30 × 10 Findings: Between December 10, 2022, and August 15, 2024, 63 patients were enrolled. All participants completed the full treatment and follow-up schedule. 52 patients (83%) reached the primary endpoint, with 46 (73%) having platelet counts of at least 50 × 10 Interpretation: These findings suggest that daratumumab could be a potential therapeutic option for adults with immune thrombocytopenia, with observations of rapid platelet recovery, durable responses, and an acceptable safety profile. Funding: The Noncommunicable Chronic Diseases-National Science and Technology Major Project, National Natural Science Foundation of China, CAMS Innovation Fund for Medical Sciences, National Key Research and Development Program of China, Tianjin Municipal Science and Technology Commission Grant, Joint Funds of the Natural Science Foundation of Tianjin, and China Higher Education Institution Industry-University-Research Innovation Fund.