title: Are we altering the natural history of primary myelofibrosis?
abstract: Primary myelofibrosis (PMF) is a clonal hematologic malignancy with a variable disease course; survival ranges from months to years. Historically, only allogeneic hematopoietic stem cell transplantation (alloHSCT) has demonstrated an ability to alter the natural history of PMF, but high treatment-related mortality risks limit the utility of alloHSCT to a minority of patients with PMF or myelofibrosis secondary to other myeloproliferative neoplasms. The recent development of therapies that regulate the Janus kinase-signal transducer and activator of transcription signaling pathway has changed the treatment landscape from primarily palliative treatment to potential disease modification.
authors.affiliation: Department of Medicine, Vanderbilt University Medical Center, Nashville, TN, USA. Electronic address: michael.savona@vanderbilt.edu.
authors.forename: Michael R
authors.initials: MR
authors.lastname: Savona
mesh_terms: D015415:Biomarkers; D018450:Disease Progression; D006801:Humans; D053614:Janus Kinase 2 / Q000037:antagonists & inhibitors / Q000502:physiology; D055728:Primary Myelofibrosis / Q000175:diagnosis / Q000628:therapy*; D011379:Prognosis; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use; D050791:STAT Transcription Factors / Q000502:physiology; D015398:Signal Transduction; D016896:Treatment Outcome
chemical_list: D015415:Biomarkers; D047428:Protein Kinase Inhibitors; D050791:STAT Transcription Factors; C507924:JAK2 protein, human; D053614:Janus Kinase 2
keywords: JAK inhibitors; Myelofibrosis; Proinflammatory cytokines; Spleen volume; Targeted therapy