Treatment Outcomes and Prognostic Factors in 66 Patients with Chronic Myelomonocytic Leukemia (CMML) in a Single Center

INTRODUCTION: Chronic myelomonocytic leukemia (CMML) is a rare hematological malignancy bearing of both myelodysplastic syndrome and myeloproliferative neoplasm characteristics. Despite the low incidence, the clinical diagnosis of CMML was difficult and the survival was poor. The optimal first-line therapy for CMML still remains a matter of debate. METHODS: We retrospectively analyzed the clinical characteristics of 66 CMML patients in a single center during the past 10 years and studied the survival status of CMML patients in the real world and the influence of treatment methods on the prognosis of patients. RESULTS: For the 66 CMML patients, the median age was 60 years old (IQR 47.0–67.0), and an approximately 1.6:1.0 male-to-female ratio was found. CMML-0, CMML-1 and CMML-2 accounted for 13.7% (9/66), 43.9% (29/66) and 42.4% (28/66), respectively. The chromosome abnormality rate was 27.2% (18/66). Gene mutation was detected in 60 patients by sequenced in first-generation with 51.1% (22/43) gene mutations and in NGS with 82.3% (14/17) gene mutations. The top three mutation genes were ASXL1(MT) (11/60, 18.3%), TET2(MT) (10/60, 16.7%), and SRSF2 (MT) (9/60, 15.0%). There were 27 patients in supportive therapy group, and 39 patients in chemotherapy group including patients undergoing HSCT. Patients in chemotherapy group showed better OS than those in the supportive group before and after PSM analysis with p < 0.05. Patients with blast cell in bone marrow ≥10% or WHO CMML-2 benefited more from chemotherapy treatment achieving better OS. Multivariate analysis showed that supportive therapy and intermediate-2/high in CPSS were independent risk factors for OS after PSM. DISCUSSION: Chemotherapy including hypomethylating agents prolonged overall survival of CMML patients, especially in patients with blast cell ≥10% in bone marrow or WHO CMML-2 comparing with supportive therapy. Sequencing may provide direct insight into the molecular mechanism by detection of gene mutation, enabling personalized treatment in the future.