N Engl J Med | Lei Zhang’s Team Reports First Clinical Study of Daratumumab in Pediatric Relapsed/Refractory Immune Thrombocytopenia, Demonstrating Significant Efficacy
Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by antibody-mediated platelet destruction and impaired platelet production, resulting in an increased risk of bleeding. In children, the annual incidence of ITP is approximately 1.9 to 6.4 per 100,000, making it the most common cause of acquired thrombocytopenia in pediatric patients. While most children achieve spontaneous remission either without treatment or following initial therapy, a subset of patients experience recurrent relapses requiring second-line interventions, including rituximab, thrombopoietin receptor agonists (TPO-RAs), splenectomy, and immunosuppressive agents. Due to the lack of prospective clinical trial data in children, selecting optimal second-line therapies remains a considerable clinical challenge.