Ruxolitinib for the treatment of patients with polycythemia vera

Polycythemia vera (PV) is a hematopoietic proliferative disorder associated with Janus-associated kinase (JAK)/signal transducer and activator of transcription (STAT) 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 to 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 2 and 3 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.