summary
Polycythemia vera (PV) is a rare and incurable blood cancer associated with an overproduction of blood cells in the bone marrow that affects roughly one to three people per 100,000 globally.1 The disease is driven by the dysregulation of the JAK-STAT pathway.2 It is typically characterized by elevated hematocrit, the volume percentage of red blood cells in whole blood, which can lead to a thickening of the blood and an increased risk of blood clots, as well as an elevated white blood cell and platelet count.2 This can cause serious cardiovascular complications, such as stroke and heart attack, resulting in increased morbidity and mortality.3 Additionally, patients with PV may have an enlarged spleen and symptoms that are frequent and burdensome, with an overall impact on quality of life similar to that seen with myelofibrosis.4 PV is traditionally managed by phlebotomy, a procedure to remove blood from the body to reduce the concentration of red blood cells, which is used to help maintain a hematocrit level below 45%.3 However, phlebotomy is usually unsuitable as a permanent treatment option due to its inability to control symptoms or effectively manage the overproduction of red blood cells, therefore cytoreductive agents, such as hydroxyurea, may be added.3 Unfortunately, approximately 25% of patients with PV become resistant to or intolerant of hydroxyurea treatment according to ELN criteria, resulting in inadequate disease control and an increased risk of progression.5 Recent advances in the management of these patients with inadequately controlled PV formed the topic of a Novartis sponsored satellite symposium hosted during the 2015 annual meeting of the European Hematology Association (EHA).
(BELG J HEMATOL 2015;6(3):121–3)
