{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,22]],"date-time":"2025-10-22T00:51:01Z","timestamp":1761094261523,"version":"build-2065373602"},"reference-count":34,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2013,1,10]],"date-time":"2013-01-10T00:00:00Z","timestamp":1357776000000},"content-version":"vor","delay-in-days":9,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["accpjournals.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pharmacotherapy"],"published-print":{"date-parts":[[2013,1]]},"abstract":"<jats:p>Myelofibrosis is a myeloproliferative neoplasm characterized by bone marrow fibrosis and extramedullary hematopoiesis. Evolution of myelofibrosis can lead to life\u2010threatening complications, including transformation to leukemia, thrombotic events, and hemorrhagic episodes. The only curative therapy for myelofibrosis is allogeneic hematopoietic stem cell transplantation. Because this disease manifests primarily in the older population, many patients diagnosed with myelofibrosis are not considered medically fit for such aggressive therapy. Other available medical therapies do not halt disease progression; instead, current treatment strategies have focused on targeting specific symptomology, although with limited efficacy. The lack of effective treatment options for patients with myelofibrosis has rendered this orphan disease state an unmet medical need, and novel approaches to improve outcomes are necessary. Emerging research has identified numerous molecular mutations in patients with myelofibrosis, making this disease a potential candidate for molecularly targeted therapy. The most prevalent mutation identified is a gain\u2010of\u2010function mutation in the Janus kinase (JAK) family, <jats:styled-content style=\"fixed-case\">JAK<\/jats:styled-content>2 <jats:styled-content style=\"fixed-case\">V<\/jats:styled-content>617<jats:styled-content style=\"fixed-case\">F<\/jats:styled-content>, which has been identified in more than half of patients with myelofibrosis. This mutation results in a constitutively active <jats:styled-content style=\"fixed-case\">JAK<\/jats:styled-content>\u2010signal transducer and activator of transcription pathway resulting in\u00a0dysregulated\u00a0cellular proliferation and hematopoiesis. Ruxolitinib is a small\u2010molecule inhibitor\u00a0of <jats:styled-content style=\"fixed-case\">JAK<\/jats:styled-content>1 and <jats:styled-content style=\"fixed-case\">JAK<\/jats:styled-content>2 and recently became the first drug approved by the <jats:styled-content style=\"fixed-case\">United States F<\/jats:styled-content>ood and <jats:styled-content style=\"fixed-case\">D<\/jats:styled-content>rug <jats:styled-content style=\"fixed-case\">A<\/jats:styled-content>dministration for the treatment of symptomatic intermediate\u2010 or high\u2010risk myelofibrosis. In clinical trials, ruxolitinib demonstrated\u00a0promising efficacy in reducing splenomegaly and myelofibrosis\u2010related symptoms. However, ruxolitinib did not demonstrate disease\u2010modifying potential and is not considered a curative\u00a0therapeutic option. Adverse events associated with\u00a0ruxolitinib are primarily hematologic, with thrombocytopenia and anemia being the most common toxicologic events identified. Future research will shed light on whether ruxolitinib in combination with other treatments will further enhance outcomes in myelofibrosis.<\/jats:p>","DOI":"10.1002\/phar.1165","type":"journal-article","created":{"date-parts":[[2013,1,10]],"date-time":"2013-01-10T08:47:27Z","timestamp":1357807647000},"page":"84-92","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":19,"title":["Ruxolitinib: A New Treatment Option for Myelofibrosis"],"prefix":"10.1002","volume":"33","author":[{"given":"Alex","family":"Ganetsky","sequence":"first","affiliation":[{"name":"Pharmacy Department Hospital of the University of Pennsylvania Philadelphia 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