Polycythemia Vera (PV): Update on Emerging Treatment Options

Abstract

Polycythemia Vera (PV) is a chronic myeloproliferative neoplasm characterized by exuberant red cell production leading to a broad range of symptoms that compromise quality of life and productivity of patients. PV reduces survival expectation, primarily due to thrombotic events, transformation to blast phase and post-PV myelofibrosis or to development of second cancers, which are associates with poor prognosis. Current therapeutic first line recommendations based on risk adapted classification divided patients into two groups, according to age (< or >60 years) and presence of prior thrombotic events. Low-risk patients (age <60 years and no prior history of thrombosis) should be treated with aspirin (81–100 mg/d) and phlebotomy, to maintain hematocrit <45%. High-risk patients (age >60 years and/or prior history of thrombosis), in addition to aspirin and phlebotomies, should receive cytoreductive therapy in order to reduce thrombotic risk. Nowadays hydroxyurea still remains the cytoreductive agent of first choice, reserving Interferon to young patients or childbearing women. During the last years, ruxolitinib emerged as a new treatment in PV patients, as second line therapy: it appeared especially effective in patients with severe pruritus, symptomatic splenomegaly, or post-PV myelofibrosis symptoms. Currently, in PV treatment, several molecules have been tested or are under investigation. At present, the drug that has shown the most encouraging results is givinostat.

Ruxolitinib (a JAK2 inhibitor) as an emerging therapy for refractory pruritis in a patient with low-risk polycythemia vera

“…However, around 25% of patients will not tolerate hydroxyurea therapy. Although our patient was on both phlebotomy and hydroxyurea therapy, pruritis did not improve at all, and his quality of life was severely affected by that itching [2,3,5,6,9] …”

“…PV patients with either age above 60 years or a history of thrombosis are classified as having “high-risk” disease, while the absence of both risk factors is considered a “low-risk” disease. This classification into low/high risk is important in PV management [2,3] . Pruritis is reported in 40% of PV patients.…”

Post-Polycythemia Vera Myelofibrosis (PPV- MF)-A Case Report of a Patient Transplanted after an Adverse Reaction to Covid Vaccination

“…Polycythemia Vera (PV) is a haematological malignancy with a relatively favourable prognosis [1]. The outcome of PV patients is influenced by various factors including the risk of lifethreatening thrombosis and the development of the disease into the plastic phase or myelofibrosis with myeloid metaplasia [2]. Myeloproliferative neoplasms (MPN) are a category of disorders arising from clonal proliferation of hematopoietic stem cells.…”

Givinostat-Liposomes: Anti-Tumor Effect on 2D and 3D Glioblastoma Models and Pharmacokinetics

“…Among HDACis, Givinostat (ITF2357) is a potent pan-HDAC inhibitor that was first described by Leoni F. et al in 2005 [ 7 ]. It has completed phase II clinical trials for polycythaemia vera [ 8 ] and is presently being evaluated in a phase III trial for Duchenne muscular dystrophy. The achievement of this advanced drug development phase was determined by the superior tolerability of Givinostat with respect to other already approved HDACis [ 9 ].…”