New drugs in essential thrombocythemia and polycythemia vera

• Published in: Blood reviews Vol. 11; no. 1; pp. 1 - 7
• Main Authors: Tefferi, A., Elliott, M.A., Solberg, L.A., Silverstein, M.N.
• Format: Journal Article
• Language: English
• Published: Kent Elsevier Ltd 01.03.1997; Elsevier
• Subjects: Antineoplastic agents; Antineoplastic Agents - therapeutic use; Biological and medical sciences; Fibrinolytic Agents - therapeutic use; Humans; Immunotherapy; Interferon-alpha - therapeutic use; Medical sciences; Pharmacology. Drug treatments; Polycythemia Vera - drug therapy; Quinazolines - therapeutic use; Recombinant Proteins; Thrombocytosis - drug therapy; Thrombocytosis - etiology; Human; Alpha interferon; Cytokine; Polycythemia vera; Hemopathy; Essential; Thrombocythemia; Myeloproliferative syndrome; Chemotherapy; Chronic; Platelet; Treatment; Immunotherapy
• ISSN: 0268-960X; 1532-1681
• DOI: 10.1016/S0268-960X(97)90001-1

Among the chronic myeloproliferative disorders, polycythemia vera and essential thrombocythemia are unique because of their association with thrombohemorrhagic manifestations and their relatively indolent clinical course. Patients with essential thrombocythemia may not have a significant shortening of life-expectancy and most may not require specific therapy. However, patients with polycythemia vera have a significant risk of transformation of polycythemia vera into acute leukemia or postpolycythemic myelofzbrosis (or both). ‘High-risk-for-thrombosis’ patients with either polycythemia vera or essential thrombocythemia require specific therapy with a platelet-lowering agent to prevent thrombotic complications. Currently, the standard agent used for this is hydroxyurea. However, its teratogenic and leukemogenic potential has been of concern. As a result, new platelet-lowering agents are being evaluated in the treatment of polycythemia vera and essential thrombocythemia. Anagrelide and interferon alfa are two such agents and have been shown to be effective in reducing platelet counts in patients with chronic myeloproliferative disorders. The putative mechanism of action of these drugs, their specific activity in polycythemia vera and essential thrombocythemia, side-effect profile, and current indications are discussed herein.