title: Ruxolitinib for the treatment of patients with polycythemia vera.
abstract: Polycythemia vera (PV) is a hematopoietic proliferative disorder associated with Janus-associated kinase/signal transducer and activator of transcription pathway dysregulation resulting in erythrocytosis and, possibly, leukocytosis and thrombocytosis. Patients diagnosed with PV experience a broad range of symptoms associated with a reduced quality of life, often develop splenomegaly, and have an increased risk of death compared with age-matched subjects without PV. Current treatment options, notably hydroxyurea, help with disease management; however, insufficient efficacy or progressive resistance occurs in some patients, highlighting the need for new treatment options. Ruxolitinib is an oral JAK1/JAK2 inhibitor that has been evaluated in Phase II and III clinical trials in patients with PV, who are intolerant of or resistant to hydroxyurea. In this setting, ruxolitinib treatment has demonstrated normalization of blood cell counts, reduction in splenomegaly and improvements in PV-related symptom burden.
authors.affiliation: Hôpital Saint-Louis and Université Paris Diderot, Paris, France.
authors.forename: Jean-Jacques
authors.initials: JJ
authors.lastname: Kiladjian
authors.forename: Elliott F
authors.initials: EF
authors.lastname: Winton
authors.forename: Moshe
authors.initials: M
authors.lastname: Talpaz
authors.forename: Srdan
authors.initials: S
authors.lastname: Verstovsek
mesh_terms: D017321:Clinical Trials, Phase I as Topic; D017322:Clinical Trials, Phase II as Topic; D017326:Clinical Trials, Phase III as Topic; D006801:Humans; D053612:Janus Kinases / Q000037:antagonists & inhibitors; D009570:Nitriles; D011087:Polycythemia Vera / Q000188:drug therapy* / Q000378:metabolism; D047428:Protein Kinase Inhibitors / Q000494:pharmacology* / Q000627:therapeutic use*; D011720:Pyrazoles / Q000494:pharmacology* / Q000627:therapeutic use*; D011743:Pyrimidines; D016896:Treatment Outcome
chemical_list: D009570:Nitriles; D047428:Protein Kinase Inhibitors; D011720:Pyrazoles; D011743:Pyrimidines; C540383:ruxolitinib; D053612:Janus Kinases
keywords: Janus kinase 1; Janus kinase 2; erythrocytosis; myeloproliferative neoplasm; polycythemia vera; ruxolitinib; splenomegaly; thrombocytosis