The recent discovery of the JAK2 mutation and its role in the pathogenesis of myeloproliferative neoplasms led to the development of a novel class of therapeutic agents, the oral JAK2 inhibitors. These agents are effective in decreasing organomegaly and ameliorating constitutional symptoms in patients with myelofibrosis, regardless of the mutational status. Among this new class of agents is pacritinib, a dual JAK2 and FLT3 inhibitor that showed evidence of clinical efficacy in early-phase trials of patients with myelofibrosis, with limited hematologic toxicity.
Keywords: FLT3 inhibitor; JAK2 inhibitor; SB1518; essential thrombocythemia; myelofibrosis; myeloproliferative neoplasms; pacritinib; polycythemia vera; ruxolitinib.