Multicenter phase 2 study of combination therapy with ruxolitinib and danazol in patients with myelofibrosis

K. Gowin, H. Kosiorek, A. Dueck, J. Mascarenhas, R. Hoffman, C. Reeder, J. Camoriano, R. Tibes, K. Gano, J. Palmer, R. Mesa

Research output: Contribution to journalArticlepeer-review

19 Scopus citations


Myelofibrosis is a myeloproliferative neoplasm that is characterized by splenomegaly, profound symptom burden, and cytopenias. JAK inhibitor therapy offers improvements in splenomegaly, symptom burden, and potentially survival; however, cytopenias remain a significant challenge. Danazol has previously demonstrated improvements in myelofibrosis-associated anemia. We conducted a phase II clinical trial evaluating the efficacy and tolerability of combination therapy with ruxolitinib, an oral JAK inhibitor, and danazol. Fourteen intermediate or high-risk MF patients were enrolled at 2 institutions. Responses per IWG-MRT criteria were stable disease in 9 patients (64.2%) clinical improvement in 3 (21.4%) all of which were spleen responses, partial response in 1 (7.1%) and progressive disease in 1 (7.1%). Despite limited IWG-MRT response, stabilization of anemia and thrombocytopenia was demonstrated. In JAK inhibitor naïve patients, 4/5 (80%) had stable or increasing hemoglobin. Of the 9 patients on prior JAK inhibitor, 5 patients (55.5%) and 8 patients (88.9%) had stable or increasing hemoglobin or platelet levels, respectively. Adverse events possibly related included grade 3 or greater hematologic toxicity in ten patients (71.4%) and non-hematologic toxicity in two patients (14.3%). Although combination therapy did not lead to increased hematologic response per IWG-MRT criteria, hematologic stabilization was observed and may be clinically useful.

Original languageEnglish (US)
Pages (from-to)31-35
Number of pages5
JournalLeukemia Research
StatePublished - Sep 2017


  • Cytopenias
  • Danazol
  • Myelofibrosis
  • Ruxolitinib

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research


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