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; aclarubicin, 5 - 7 mg×m(-2)×d(-1) intravenously, 1 - 8 d, 15 - 23 d; cytarabine 15 mg/m(2) 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.