{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,31]],"date-time":"2026-10-31T17:26:00Z","timestamp":1793467560219,"version":"4.5.0"},"reference-count":15,"publisher":"American Society of Hematology","issue":"24","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2018,12,26]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>Myeloproliferative neoplasms (MPN), including polycythemia vera, essential thrombocythemia, and primary myelofibrosis, have a propensity to evolve into accelerated and blast-phase disease (MPN-AP\/BP), carrying a dismal prognosis. Conventional antileukemia therapy has limited efficacy in this setting. Thus, MPN-AP\/BP is an urgent unmet clinical need. Modest responses to hypomethylating agents and single-agent ruxolitinib have been reported. More recently, combination of ruxolitinib and decitabine has demonstrated synergistic in vitro activity in human and murine systems. These observations led us to conduct a phase 1 study to explore the safety of combined decitabine and dose-escalated ruxolitinib in patients with MPN-AP\/BP. A total of 21 patients were accrued to this multicenter study. Ruxolitinib was administered at doses of 10, 15, 25, or 50 mg twice daily in combination with decitabine (20 mg\/m2 per day for 5 days) in 28-day cycles. The maximum tolerated dose was not reached. The most common reasons for study discontinuation were toxicity\/adverse events (37%) and disease progression (21%). Fourteen patients died during study treatment period or follow-up. The median overall survival for patients on study was 7.9 months (95% confidence interval, 4.1-not reached). Among evaluable patients, the overall response rate by protocol-defined criteria (complete remission with incomplete count recovery + partial remission) was 9\/17 (53%) and by intention-to-treat analysis was 9\/21 (42.9%). The combination of decitabine and ruxolitinib was generally well tolerated by patients with MPN-AP\/BP and demonstrates potentially promising clinical activity. A phase 2 trial evaluating the efficacy of this combination regimen is ongoing within the Myeloproliferative Disorder Research Consortium.<\/jats:p>","DOI":"10.1182\/bloodadvances.2018019661","type":"journal-article","created":{"date-parts":[[2018,12,18]],"date-time":"2018-12-18T21:55:13Z","timestamp":1545170113000},"page":"3572-3580","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":61,"title":["Safety and efficacy of combined ruxolitinib and decitabine in accelerated and blast-phase myeloproliferative neoplasms"],"prefix":"10.1182","volume":"2","author":[{"given":"Raajit K.","family":"Rampal","sequence":"first","affiliation":[{"name":"Leukemia Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John O.","family":"Mascarenhas","sequence":"additional","affiliation":[{"name":"Tisch Cancer Institute, Icahn School of Medicine at 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