Hydroxyurea: the comparator in studies with new anti-JAK2 inhibitors


https://doi.org/10.4081/hmr.v3i3.584
Vol. 3 No. 3: 7th Meeting New Insights in Hematology Venice, Italy, 26-29 April 2009
Published: June 12, 2009
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PDF: 504
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The fundamental discovery of V617F and other mutations in the JAK2 gene of most patients with Philadelphia-negative chronic myelo-proliferative neoplasms (MPNs) has changed the diagnostic approach to these disorders and opened the way to a molecularlytargeted therapy.1-6 A new generation of drugs with more or less specific inhibitory activity against JAK2 has been developed and it is now in advanced stages of clinical evaluation.7 As a rule, the efficacy and safety of a new therapeutic agent in a given disease should be compared with the best available treatment in randomized phase III clinical trials. In the most frequent MPNs, that are essential thrombocythemia (ET) and polycythemia vera (PV), the standard myelosuppressive therapy for patients with a high risk of thrombosis is hydroxyurea (HU). This drug is also the first choice for patients with myelofibrosis (MF) and symptomatic splenomegaly or requiring cytoreduction. The purpose of this chapter is to review the expected benefits and risks of HU in patients with MPNs in order to establish the bottom line for comparative studies with new drugs.

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Finazzi, G., & Barbui, T. (2009). Hydroxyurea: the comparator in studies with new anti-JAK2 inhibitors. Hematology Meeting Reports (formerly Haematologica Reports), 3(3). https://doi.org/10.4081/hmr.v3i3.584
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