title: Myelofibrosis: an update on drug therapy in 2016.
abstract: Primary myelofibrosis (PMF) is the least common but the most aggressive of the classic Philadelphia chromosome-negative myeloproliferative neoplasms. Survival is much shorter in PMF than in polycythemia vera (PV) or essential thrombocythemia (ET). Post-PV/ET myelofibrosis (MF) is clinically indistinguishable from PMF and approached similarly. Areas covered: Current pharmacologic therapy of MF revolves around the Janus kinase 1/2 (JAK1/2) inhibitor ruxolitinib, which dramatically improves constitutional symptoms and splenomegaly in the majority of patients, and improves overall survival (OS). However, allogeneic stem cell transplantation remains the only potential cure. Other JAK inhibitors continue to be developed for MF, and momelotinib and pacritinib are in phase III clinical trials. Anemia is common in MF, and initially worsened by ruxolitinib. Momelotinib and pacritinib may prove advantageous in this regard. Current strategies for managing anemia of MF include danazol, immunomodulatory drugs and erythroid stimulating agents, either alone or in combination with ruxolitinib. Expert opinion: A number of other agents, representing diverse drug classes, are in various stages of development for MF. These include newer JAK inhibitors, other signaling inhibitors, epigenetic modifiers, anti-fibrotic agents, telomerase inhibitors, and activin receptor ligand traps (for anemia). Hopefully, these novel therapies will further extend the clinical benefits of ruxolitinib.
authors: a Department of Leukemia , University of Texas MD Anderson Cancer Center , Houston , TX , USA.
authors: Prithviraj
authors: P
authors: Bose
authors: a Department of Leukemia , University of Texas MD Anderson Cancer Center , Houston , TX , USA.
authors: Srdan
authors: S
authors: Verstovsek
mesh_terms: D000818:Animals; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D006801:Humans; D053613:Janus Kinase 1 / Q000037:antagonists & inhibitors / Q000378:metabolism; D053614:Janus Kinase 2 / Q000037:antagonists & inhibitors / Q000378:metabolism; D055728:Primary Myelofibrosis / Q000175:diagnosis* / Q000188:drug therapy* / Q000201:enzymology; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use*
chemical_list: D000970:Antineoplastic Agents; D047428:Protein Kinase Inhibitors; C507904:JAK1 protein, human; C507924:JAK2 protein, human; D053613:Janus Kinase 1; D053614:Janus Kinase 2
keywords: Imids; JAK inhibitors; Myelofibrosis; danazol; erythroid stimulating agents; hydroxyurea; hypomethylating agents; momelotinib; pacritinib; ruxolitinib