1) Define assessment of prognosis in Myelofibrosis
2) Discuss role of new therapies vs. conventional therapies in Myelofibrosis
3) Define methods for creating an individualized plan for myelofibrosis patients
Myelofibrosis (MF) is a potentially fatal myeloproliferative neoplasm (MPN) with a very heterogeneous group of patients with widely variable prognosis. Clinical management options range from low-risk/low-reward observation to high-risk/high-reward allogeneic stem cell transplantation. Medical therapy ranges from off label utilization of palliative agents for MF associated anemia or splenomegaly; however, none of these latter agents impact the natural history of the illness. The discovery of the JAK2-V617F, and subsequent additional MPN associated mutations has led to development of a spectrum of selective inhibitors of JAK2. Clinical trials with these latter agents have led to meaningfully reductions in MF-associated splenomegaly and constitutional symptoms. Several additional therapies that do not directly target JAK2 (e.g., immunomodulatory drugs, histone deacetylase inhibitors) may ameliorate MF-associated anemia and morbidity-inducing symptoms. Balancing the potential benefits of these new agents against the risks and benefits of allogeneic stem cell transplantation requires an accurate estimation of the prognosis for an individual patient and development of individualized treatment plans. Evolving information regarding the efficacy of new medicines (alone or in consolidation) will continue to modify MF treatment strategies and plans.
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