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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.

Original publication

DOI

10.1080/10428194.2016.1195501

Type

Journal article

Journal

Leuk Lymphoma

Publication Date

10/2016

Volume

57

Pages

2259 - 2267

Keywords

Intermediate-1 risk, International Prognostic Scoring System, myelofibrosis, ruxolitinib, splenomegaly, Clinical Trials as Topic, Combined Modality Therapy, Hematopoietic Stem Cell Transplantation, Humans, Nitriles, Patient Selection, Primary Myelofibrosis, Prognosis, Protein Kinase Inhibitors, Pyrazoles, Pyrimidines, Risk, Treatment Outcome