Novel therapeutic strategies for essential thrombocythemia/polycythemia vera

Abstract

Myeloproliferative neoplasms (MPNs) are clonal disorders of hematopoietic stem cells; these include polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF). MPNs are inflammatory cancers, wherein the malignant clone generates cytokines that sustain the inflammatory drive in a self-perpetuating vicious cycle. The course of MPNs follows a biological continuum, that is, from early cancer stages (ET/PV) to advanced myelofibrosis as well as impending leukemic transformation. MPN-related symptoms, e.g., fatigue, general weakness, and itching, are caused by inflammatory cytokines. Thrombosis and bleeding are also exacerbated by inflammatory cytokines in patients with MPN. Until recently, the primary objective of ET and PV therapy was to increase survival rates by preventing thrombosis. However, several medications have recently demonstrated the ability to modify the course of the disease; symptom relief is expected for most patients. In addition, there is increasing interest in the active treatment of patients at low risk with PV and ET. This review focuses on the ET/PV treatment strategies as well as novel treatment options for clinical development.

Treatment with ropeginterferon alfa-2b in patients with hydroxyurea resistant or intolerant polycythemia vera in South Korea: one-year results from a phase 2 study

“…Yet, the implementation of these treatments is hindered in Korea due to a lack of reimbursement. Furthermore, most research on interferon in PV has focused on its use as an early-stage treatment option due to its disease-modifying properties [ 8 , 9 ]. Consequently, data on the clinical efficacy and safety of interferon in patients resistant or intolerant to HU are sparse, particularly regarding treatment with ropeginterferon alfa-2b.…”