Phase 2 study of the JAK kinase inhibitor ruxolitinib in patients with refractory leukemias, including postmyeloproliferative neoplasm acute myeloid leukemia

Alireza Eghtedar, Srdan Verstovsek, Zeev Estrov, Jan Burger, Jorge Cortes, Carol Bivins, Stefan Faderl, Alessandra Ferrajoli, Gautam Borthakur, Solly George, Peggy A. Scherle, Robert C. Newton, Hagop M. Kantarjian, Farhad Ravandi

Research output: Contribution to journalArticle

131 Scopus citations

Abstract

We conducted a phase 2 study of ruxolitinib in patients with relapsed/refractory leukemias. Patients with acceptable performance status (0-2), adequate organ function, and no active infection, received ruxolitinib 25 mg orally twice a day for 4 weeks (1 cycle). Response was assessed after every 2 cycles of treatment, and patients who completed 2 cycles were allowed to continue treatment until disease progression. Dose escalation to 50 mg twice daily was permitted in patients demonstrating a benefit. Thirtyeight patients, with a median age of 69 years (range, 45-88), were treated. The median number of prior therapies was 2 (range, 1-6). Twelve patients had JAK2V617F mutation. Patients received a median of 2 cycles of therapy (range, 1-22). Three of 18 patients with post-myeloproliferative neoplasm (MPN) acute myeloid leukemia (AML) showed a significant response; 2 achieved complete remission (CR) and one achieved a CR with insufficient recovery of blood counts (CRi). The responding patients with palpable spleens also had significant reductions in spleen size. Overall, ruxolitinib was very well tolerated with only 4 patients having grade 3 or higher toxicity. Ruxolitinib has modest antileukemic activity as a single agent, particularly in patients with post-MPN AML. The study was registered at www.clinicaltrials.gov as NCT00674479.

Original languageEnglish (US)
Pages (from-to)4614-4618
Number of pages5
JournalBlood
Volume119
Issue number20
DOIs
StatePublished - May 17 2012

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

Fingerprint Dive into the research topics of 'Phase 2 study of the JAK kinase inhibitor ruxolitinib in patients with refractory leukemias, including postmyeloproliferative neoplasm acute myeloid leukemia'. Together they form a unique fingerprint.

  • Cite this

    Eghtedar, A., Verstovsek, S., Estrov, Z., Burger, J., Cortes, J., Bivins, C., Faderl, S., Ferrajoli, A., Borthakur, G., George, S., Scherle, P. A., Newton, R. C., Kantarjian, H. M., & Ravandi, F. (2012). Phase 2 study of the JAK kinase inhibitor ruxolitinib in patients with refractory leukemias, including postmyeloproliferative neoplasm acute myeloid leukemia. Blood, 119(20), 4614-4618. https://doi.org/10.1182/blood-2011-12-400051