The efficacy and safety of valproic acid in combination with low dose chemotherapy on intermediate and high-risk myelodysplastic syndrome / 中华内科杂志

ABSTRACT

Objective To evaluate the efficacy and adverse effect of valproic acid (VPA) in combination with low dose chemotherapy on intermediate and high-risk myelodysplastic syndrome. Methods A total of 41 patients with intermediate (34) and high-risk (7) myelodysplastic syndrome were retrospectively analyzed. Among them, 19 patients received low dose chemotherapy regimen and 22 received low dose chemotherapy plus VPA.Low dose chemotherapy regimen included homoharringtonine,1-2 mg·m-2·d-1 intravenously,14-28 d; clarubicin,5-7 mg·m-2·-1 intravenously,1-8 d,15-23 d;cytarabine 15 mg/m2 subcutaneously once every 12 h, 14-21 d; and subcutaneously use of granulocyte colony-stimulating factor 200 μg·m-2·d -1 when neutrophil deficiency.The outcome and adverse effect were recorded after the treatment. Results The overall response rate in the low dose chemotherapy regimen group was 47.4% (9/19), 6 patients (31.6%) achieved complete response (CR). The overall response rate in the VPA group was 77.2% (17/22), 9 patients (40.9%) achieved CR. The overall response rate of the low dose chemotherapy in combination with VPA group was significantly higher than that in the low dose chemotherapy group (P＜0.05) while no difference was found in CR rate. The adverse effect of the low dose chemotherapy in combination with VPA regimen was tolerated. Conclusion With acceptable adverse effect, the low dose chemotherapy in combination with VPA regimen is effective for the treatment of intermediate and high-risk myelodysplastic syndrome. Long-term outcome needs further investigation.