[The application of thrombopoietin receptor agonists in the treatment of non-muscle myosin heavy chain 9-related disease].

MYH9-related disease (MYH9-RD) is an autosomal dominant disorder caused by mutations in the MYH9 gene, characterized by macrothrombocytopenia, neutrophil inclusions, and other features. Due to its low incidence and nonspecific clinical manifestations, many patients are misdiagnosed or undiagnosed. Clinical management often includes platelet transfusion, pro-hemostatic or antifibrinolytic agents, avoidance of antiplatelet drugs, and hemorrhage prevention. Hematopoietic stem cell transplantation may be a treatment option for severe cases. For a long time, platelet transfusion has been the most commonly used treatment for preventing or managing bleeding in MYH9-RD patients, but it is associated with various adverse reactions and clinical limitations. In recent years, thrombopoietin receptor agonists (TPO-RA), as a novel therapeutic class, have been increasingly used in MYH9-RD and have demonstrated promising efficacy in increasing platelet counts and reducing bleeding symptoms. However, their mechanisms of action, clinical effectiveness, and safety profiles require further investigation. This article aims to review the application of TPO-RA in MYH9-RD, analyze their mechanisms, clinical outcomes, and future research directions, thereby providing insights and references for further studies in this field.