Myelofibrosis (MF) is a rare, chronic myeloproliferative neoplasm (MPN).1 MF may occur in the absence of known pre-existing conditions as it does in primary MF (PMF) or may develop secondarily from polycythemia vera (PV) or essential thrombocythemia (ET).1 PMF, post-PV, and post-ET MF (collectively called MPN-associated MF) are types of classical MPN that lack BCR-ABL1 translocations (excluding them from chronic myeloid leukemia diagnoses) and are characterized by bone marrow stem cell dysfunctions.2,3 PMF is the most common form of MPN-associated MF and generally occurs in adults of advanced age.4,5 Up to 95% of patients may present with at least two MF-related symptoms, such as anemia, leukopenia, thrombocytopenia, weight loss, fatigue, and abdominal pain associated with splenomegaly.6 Overactive cytokine signaling may cause systemic inflammation and alterations of the bone marrow microenvironment, which may lead to inefficient hematopoiesis and extramedullary hematopoiesis.1 Somatic mutations in genes involved in key signaling pathways may drive cytokine over-signaling in MF, resulting in myeloproliferation, inflammation, and bone marrow remodeling.1,7,8
Conventional therapeutic approaches have improved outcomes for some patients with MF.9 Research areas in MF include identifying new treatment options since current standard therapeutic strategies may include 1 US Food and Drug Administration–approved treatment and supportive care therapies (eg, erythropoietin-stimulating agents, androgens, and immunomodulators).8-10 Investigational therapies include targeted therapies (eg, inhibitors of Janus kinase 1 and 2, hedgehog, and mechanistic target of rapamycin), epigenetic regulators (eg, histone deacetylase inhibitors, hypomethylating agents, and telomerase inhibitors), ligand traps, and anti-fibrotic medications, among others.11,12
The safety and efficacy of the agents and/or uses under investigation have not been established. There is no guarantee that the agents will receive health authority approval or become commercially available in any country for the uses being investigated.
Clinical trial information is sourced from ClinicalTrials.gov. Information is updated manually as clinical trials are published. The efficacy and safety of the agents and/or uses under investigation have not been established. There is no guarantee that the agents will receive health authority approval or become commercially available in any country for the uses being investigated.