Analysis on clinical efficacy and influencing factors of decitabine-based regimens in patients with myelodysplastic syndrome-refractory anemia with excess blasts

Abstract

Objective To analyze the clinical efficacy, safety and influencing factors of decitabine (DAC)-based regimens in patients with myelodysplastic syndrome-refractory anemia with excess blasts (MDS-RAEB). Methods We performed a retrospective analysis of 63 patients with MDS-RAEB treated with DAC, evaluated the clinical efficacy and adverse reactions, and analyzed the influencing factors affecting survival. Results Among 63 patients, 23 were RAEB-1 and 40 were RAEB-2. The median treatment was 4 (2-13) courses. The total effective rate of DAC for MDS-RAEB was 58.7%(37/63), and the complete response rate was 20.6%(13/63). Among 37 patients who were effective, 20 (54.1%) patients performed efficacy after 2 courses. The median course of treatment to achieve the best effect was 3.5 (3-4) courses. With a median follow-up of 14 (2-68) months, 63 patients had a overall survival rate (OS) of 84.2% and a 1-year progression-free survival rate (PFS) of 73%. In univariate analysis, the factors that prolonged OS were that the best effect after medication was stable disease (SD) (to achieve complete remission, partial remission, complete bone marrow remission, hematological improvement) (P=0.009) and no thrombocytopenia at first diagnosis (P=0.019), the factor that prolongs PFS is the best effect above SD (P=0.003). Multivariate analysis suggested that the factors affecting OS and PFS were the best curative effects above SD (P=0.015 vs P=0.008). The adverse effects of decitabine in the treatment of MDS-RAEB were mainly bone marrow suppression and pulmonary infection. Conclusions Decitabine is an effective and well-tolerated drug in the treatment of MDS-RAEB. Response to decitabine treatment is one of the independent factors affecting the prognosis. Key words: Anemia, refractory, with excess of blasts; Drug therapy, combination; Decitabine; Prognosis; Factor analysis, statistical