Ruxolitinib combined with prednisone, thalidomide and danazol in patients with myelofibrosis: Results of a pilot study

摘要

Abstract Ruxolitinib is a safe and effective therapy of myeloproliferative neoplasm‐associated (MPN) myelofibrosis. However, often there are dose reductions and/or therapy interruptions because of therapy‐related adverse events (AEs), especially anemia and thrombocytopenia. We previously reported combined therapy with prednisone, thalidomide and danazol (PTD) reversed anemia and thrombocytopenia in people with MPN‐associated myelofibrosis. We wondered whether adding PTD to ruxolitinib might mitigate the hematologic AEs and thereby avoid the dose reduction of ruxolitinib and improve the efficacy. To test this hypothesis, we conducted a baseline hemoglobin and platelet concentration assignment prospective observational study in 72 patients comparing 3‐month dose adjustment and efficacy of ruxolitinib with (N?=?53, the study group) or without (N?=?19, the control group) PTD. According to the platelet counts, the median daily ruxolitinib doses in the study group increased from 30 to 40?mg by week 12, whereas in the control group it remained at 30?mg (p?=?0.019). In the study group 35 patients had a hemoglobin increase ≥10?g/L compared with no patient receiving ruxolitinib only (p?100?×?10E+9/L were seen in 56.6% and 5.3% of patients in the two groups, respectively (p?