Efficacy and safety of combined decitabine and ruxolitinib in the treatment of chronic myelomonocytic leukemia*

Abstract

Abstract Objective The aim of the study was to evaluate the clinical efficacy of decitabine (DEC) combined with ruxolitinib (RUX) in the treatment of chronic myelomonocytic leukemia (CMML). Methods The clinical characteristics of 12 patients with CMML were analyzed retrospectively and subsequent target sequencing was performed to investigate the efficacy of the combined treatment with DEC and RUX and the molecular signatures therein. Results Among the 12 cases, clinical improvement was observed in all patients (100%), spleen reduction was observed in six patients (67%), and hematologic improvement was observed in four patients (33%). In the CMML-1 group, the overall response was 50% (3/6), one case achieved complete response, one achieved bone marrow remission, and one achieved hematological improvement. In the CMML-2 group, the overall response was 17% (1/6), one case achieved complete response, four showed disease progression (PD), and one exhibited no response. As expected, ASXL1 mutation was predictive for the outcome of CMML (hazard ratio of 2.97, 95% confidence interval of 1.21-7.06; P = 0.02). Conclusion The use of DEC combined with RUX in the treatment of CMML effectively improved the clinical response and quality of life, especially for CMML-1 patients. Ongoing clinical trials will further evaluate the safety and efficacy of this novel therapeutic approach.