Polycythemia Vera Overview

Polycythemia vera (PV), myelofibrosis and essential thrombocythemia are classified as Philadelphia chromosome-negative myeloproliferative neoplasms.1 PV is characterized by clonal stem cell proliferation of the erythroid, myeloid and megakaryocytic lines while its predominant characteristic is an increase in red blood cell mass, increased white blood cell and platelet counts are common.2,3

Polycythemia Vera (PV) is a myeloproliferative neoplasm (MPN), most often associated with the JAK2V617F point mutation that results in overactive signaling through the JAK-STAT pathway.4-6 PV is characterized by hyperproliferation of red blood cells (erythrocytosis) and significant symptom burden. 5-9 The increase in red blood cell mass results in hyperviscosity of the blood, increased thrombotic risk, significant morbidity and a shortened life expectancy.10 Advanced PV is frequently characterized by palpable splenomegaly and severe constitutional symptoms, including fatigue (85%), pruritus (65%), night sweats (49%) and bone pain (43%).11

The current therapeutic approach in PV focuses on lowering the risk for thrombotic events without exposing patients to increased risk of leukemic transformation.12 While phlebotomy and low-dose aspirin are accepted as the standard of care for initial therapy, cytoreductive therapy is recommended to aid in the control of erythrocytosis in the presence of poor tolerance to phlebotomy, symptomatic or progressive splenomegaly or evidence of high thrombotic risk. Hydroxyurea (HU) is frequently the cytoreductive agent of choice and therapeutic options beyond HU are limited.3,12


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