Phase 2 study of ruxolitinib and decitabine in patients with myeloproliferative neoplasm in accelerated and blast phase

John O. Mascarenhas, Raajit K. Rampal, Heidi E. Kosiorek, Rupali Bhave, Elizabeth Hexner, Eunice S. Wang, Aaron Gerds, Camille N. Abboud, Marina Kremyanskaya, Dimitry Berenzon, Olatoyosi Odenike, Noushin Farnoud, Aishwarya Krishnan, Rona Singer Weinberg, Erin McGovern, Mohamed E. Salama, Vesna Najfeld, Juan S. Medina-Martinez, Juan E. Arango Ossa, Max F. LevineYangyu Zhou, Lonette Sandy, Mark L. Heaney, Ross L. Levine, Ruben A. Mesa, Amylou C. Dueck, Ronald Hoffman

Research output: Contribution to journalArticlepeer-review

4 Scopus citations


Myeloproliferative neoplasms (MPN) that have evolved into accelerated or blast phase disease (MPN-AP/BP) have poor outcomes with limited treatment options and therefore represent an urgent unmet need. We have previously demonstrated in a multicenter, phase 1 trial conducted through the Myeloproliferative Neoplasms Research Consortium that the combination of ruxolitinib and decitabine is safe and tolerable and is associated with a favorable overall survival (OS). In this phase 2 trial, 25 patients with MPN-AP/BP were treated at the recommended phase 2 dose of ruxolitinib 25 mg twice daily for the induction cycle followed by 10 mg twice daily for subsequent cycles in combination with decitabine 20 mg/m2 for 5 consecutive days in a 28-day cycle. Nineteen patients died during the study follow-up. The median OS for all patients on study was 9.5 months (95% confidence interval, 4.3-12.0). Overall response rate (complete remission 1 incomplete platelet recovery 1 partial remission) was 11/25 (44%) and response was not associated with improved survival. We conclude that the combination of decitabine and ruxolitinib was well tolerated, demonstrated favorable OS, and represents a therapeutic option for this high-risk patient population. This trial was registered at as #NCT02076191.

Original languageEnglish (US)
Pages (from-to)5246-5256
Number of pages11
JournalBlood Advances
Issue number20
StatePublished - Oct 26 2020

ASJC Scopus subject areas

  • Hematology


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