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

K Gowin, Krisstina Gowin, Heidi Kosiorek, H Kosiorek, A Dueck, Amylou C. Dueck, J Mascarenhas, John Mascarenhas, Ronald Hoffman, R Hoffman, Craig B. Reeder, C Reeder, John Camoriano, J Camoriano, Raoul Tibes, R Tibes, K Gano, Katherine Gano, J Palmer, Jeanne Palmer, R Mesa, Ruben A. Mesa
Leukemia research · 2017 · vol. 60 , pp. 31–35 · doi:10.1016/j.leukres.2017.06.005 · PMID:28646676 · W2626609447
Non-randomised trial OA: closed CC0 ⤵ 2 in-corpus citations
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This multicenter phase 2 study investigated the combination therapy of ruxolitinib and danazol in patients with myelofibrosis, evaluating its efficacy and safety.

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Abstract

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.

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MeSH descriptors

Danazol Drug Therapy, Combination Primary Myelofibrosis Pyrazoles Adult Aged Anemia Anemia Danazol Danazol Drug Therapy, Combination Female Humans Male Middle Aged Nitriles Primary Myelofibrosis Primary Myelofibrosis Pyrazoles Pyrazoles

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