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Ref Type Journal Article
PMID (22474318)
Authors Mascarenhas J, Hoffman R
Title Ruxolitinib: the first FDA approved therapy for the treatment of myelofibrosis.
Journal Clinical cancer research : an official journal of the American Association for
Vol 18
Issue 11
Date 2012 Jun 1
URL
Abstract Text The BCR-ABL1-negative myeloproliferative neoplasms (e.g., essential thrombocythemia, polycythemia vera, and primary myelofibrosis) are a group of heterogeneous hematologic malignancies that involve a clonal proliferation of hematopoietic stem cells. Thrombosis, bleeding, and transformation to acute leukemia reduce the overall survival of patients with myelofibrosis, a disease typified by progressive splenomegaly and disease-related symptoms such as fatigue, pruritus, and bony pains. Hematopoietic stem cell transplant offers the only potential for cure in a minority of eligible patients, leaving a serious unmet need for improved therapies. Recent advances in our understanding of the pathogenetic mechanisms underlying these diseases have led to an explosion of clinical trials evaluating novel therapies. The discovery of an activating mutation in the Janus-activated kinase 2 (JAK2) gene provided a therapeutic target to downregulate this activated signaling pathway, which influences the phenotype of these diseases. Ruxolitinib (Jakafi; Incyte) is a small-molecule inhibitor of JAK1/2 that has proved to be effective at reducing splenomegaly and ameliorating symptoms in myeloproliferative neoplasms. Based on the results of 2 pivotal randomized phase III clinical trials, ruxolitinib has become the first therapeutic to be approved by the U.S. Food and Drug Administration for treatment of patients with myelofibrosis. Ruxolitinib offers a well-tolerated oral therapeutic option for patients with myelofibrosis with symptomatic splenomegaly and debilitating disease-related symptoms, but it does not seem to be effective at eliminating the underlying hematological malignancy.

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Ruxolitinib Ruxolitinib 36 37
Drug Name Trade Name Synonyms Drug Classes Drug Description
Ruxolitinib Jakafi INCB 18424|INCB018424|INCB18424 JAK1 Inhibitor - ATP competitive 4 JAK2 Inhibitor - ATP competitive 14 Jakafi (ruxolitinib) is an inhibitor of protein tyrosine kinases JAK1 and JAK2, thus resulting in reduced inflammation and reduced proliferation (PMID: 22474318). Jakafi (ruxolitinib) is FDA approved to treat bone marrow cancer, specifically intermediate or high-risk myelofibrosis (FDA.gov)
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown myelofibrosis not applicable Ruxolitinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Jakafi (ruxolitinib) resulted in a decrease in spleen volume of greater than or equal to 35% in 41.9% of patients with intermediate or high-risk myelofibrosis, compared to 0.7% of patients treated with the placebo (PMID: 22474318). detail... 22474318