Ruxolitinib for treatment of polycythemia vera

Introduction: The myeloproliferative neoplasm, polycythemia vera (PV) significantly impacts the quality of life of afflicted patients. The traditional therapies of phlebotomy, aspirin and cytoreductive medicines center on decreasing the risk of vascular events, but can be inadequate to improve symptom burden. Over time, these therapies may become ineffective or patients may develop intolerances providing a need for more therapeutic options. Areas covered: Recently, the randomized Phase III RESPONSE trial has demonstrated the ability to improve hematocrit control, reduce splenomegaly and improve symptoms in patients with PV. This has led to the US FDA and the European Commission approval of ruxolitinib for patients with PV who have an inadequate response or intolerance to hydroxyurea (HU). The RELIEF trial recently showed a trend for improved symptom control when ruxolitinib replaced HU in patients attaining good disease control, but inadequate symptom control on HU. Expert opinion: Ruxolitinib offers another option for therapy in PV with efficacy in hematocrit control, reducing splenomegaly and symptom burden. Future goals include knowing at what juncture in PV should Janus kinase 2 inhibitors be introduced in PV.