Advances in polycythemia vera treatment with targeted therapies and clinical trials

Abstract

Polycythemia vera (PV) is a chronic myeloproliferative disorder characterized by excessive red blood cell production, leading to a heightened risk of thrombosis, hemorrhage, and progression to myelofibrosis. While traditional therapies such as phlebotomy and hydroxyurea have been used for disease management, they do not address the underlying pathophysiology or alleviate common symptoms like fatigue and pruritus. Recent advances in targeted therapies offer promising new options for PV treatment. This review explores novel therapeutic approaches currently under investigation, including hepcidin agonists, MDM2 inhibitors, histone deacetylase (HDAC) inhibitors, and LSD1 inhibitors. These therapies aim to correct hematopoietic dysregulation, reduce symptom burden, and improve long-term outcomes for PV patients. While clinical trials show encouraging results, further studies are needed to fully evaluate their safety, efficacy, and potential for broad clinical use. Ultimately, these emerging treatments could reshape the landscape of PV management by offering more personalized, effective options for patients.