title: Ruxolitinib is effective in patients with intermediate-1 risk myelofibrosis: a summary of recent evidence.
abstract: Ruxolitinib is the only therapy with an approved indication for myelofibrosis (MF), a myeloproliferative neoplasm associated with progressive bone marrow fibrosis and extramedullary hematopoiesis. Although the pivotal phase 3 COMFORT studies included only patients with intermediate-2 or high-risk MF, the US indication includes all patients with intermediate- or high-risk disease. Data from recent nonrandomized studies confirm that the benefits of ruxolitinib established in the COMFORT studies in terms of spleen size reduction and symptom improvement also extend to patients with intermediate-1 risk MF, who tend to have less advanced disease than patients with higher-risk MF. Given the disease-modifying potential of ruxolitinib therapy, timely initiation of ruxolitinib therapy may not only improve patients' current clinical status but also lead to better long-term outcomes. The decision of whether or when to initiate ruxolitinib treatment should be based on the expected benefit-risk ratio for each patient, specifically considering potential adverse effects.
authors.affiliation: a Department of Haematology , Guy's and St. Thomas' NHS Foundation Trust , London , UK ;
authors.forename: Claire N
authors.initials: CN
authors.lastname: Harrison
authors.affiliation: b Comprehensive Cancer Center , University of Michigan , Ann Arbor , MI , USA ;
authors.forename: Moshe
authors.initials: M
authors.lastname: Talpaz
authors.affiliation: c MRC Weatherall Institute of Molecular Medicine and NIHR Biomedical Research Centre , University of Oxford , Oxford , UK.
authors.forename: Adam J
authors.initials: AJ
authors.lastname: Mead
mesh_terms: D002986:Clinical Trials as Topic; D003131:Combined Modality Therapy; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D009570:Nitriles; D018579:Patient Selection; D055728:Primary Myelofibrosis / Q000175:diagnosis* / Q000188:drug therapy* / Q000209:etiology; D011379:Prognosis; D047428:Protein Kinase Inhibitors / Q000494:pharmacology / Q000627:therapeutic use*; D011720:Pyrazoles / Q000494:pharmacology / Q000627:therapeutic use*; D011743:Pyrimidines; D012306:Risk; D016896:Treatment Outcome
chemical_list: D009570:Nitriles; D047428:Protein Kinase Inhibitors; D011720:Pyrazoles; D011743:Pyrimidines; C540383:ruxolitinib
keywords: Intermediate-1 risk; International Prognostic Scoring System; myelofibrosis; ruxolitinib; splenomegaly