Ruxolitinib in clinical practice for therapy of myelofibrosis: Single USA center experience following Food and Drug Administration approval

摘要

Myelofibrosis is a myeloproliferative neoplasm (MPN) characterized by increased bone marrow fibrosis, abnormal blood counts with peripheral cytopenias, extramedullary hematopoiesis, splenomegaly and profound constitutional symptoms [1,2]. Presenting as a primary disease or arising from polycythemia vera or essential thrombocythemia, myelofibrosis incurs a significant symptom burden for patients, and results in a reduced survival of 2-11 years [3-7]. Amongst MPNs, myelofibrosis has represented a unique challenge to efforts aimed at palliating symptoms or impacting disease course. The 2005 discovery of the gain-of-function JAK2V617F mutation present in a significant proportion of patients with polycythemia vera, essential thrombocythemia and myelofibrosis opened a new avenue of investigation into potential novel therapies [8,9]. Among the various JAK2 inhibitor agents that have been developed, ruxolitinib was the first to receive Food artd Drug Administration (FDA) approval in the USA for the treatment of intermediate- and high-risk myelofibrosis.