Objective To analyze the clinical efficacy and safety of compound Qinghuang powder (compound QHP) for treatment of myelodysplastic syndromes (MDS) and its association with blood arsenic concentration (BAC). Methods 40 patients with MDS were treated with compound QHP, and the clinical efficacy, safety, and its association with BAC were evaluated after treatment for 6, 9 months, respectively. Results After treatment for 6 months, the rate of hematology improvement was 32.5% (13/40), and the effective rate was 87.5% (35/40). 21 cases depended on the blood transfusion before treatment, after treatment 6 cases completely got rid of blood transfusion and the blood transfusion of another 6 cases was decreased by more than 50%. The absolute neutrophil count was increased from (0.50±0.13)×109/L to (0.93±0.33)×109/L (t= 4.130, P= 0.000 8). The hemoglobin content was increased from (71.06±14.82) g/L to (80.41±27.35) g/L (t= 2.233, P= 0.032 1). After treatment for 9 months, 76.2% (16/40) of the patients got rid of blood transfusion or blood transfusion reduction was more than 50%. The platelet count was increased from (45.04±24.38)×109/L to (60.65±29.46)×109/L (t= 2.241, P= 0.033 5). The incidence of abdominal pain and diarrhea after treatment for 1, 3 and 6 months were 12.5% (5/40), 10.0% (4/40) and 5.0% (2/40), respectively, all belonging to mild level. Before treatment, there were 12 patients with abnormal liver function, including 6 cases back to normal after treatment, and 6 cases of significantly relieved, without new case with abnormal liver function. Before treatment, there were 10 cases with abnormal myocardial enzymes, including 1 cases back to normal after treatment and 9 cases significantly relieved, without new case with abnormal myocardial enzymes. No patient with abnormal renal function was observed before and after treatment. The BAC was (7.71±5.65) μg/L before treatment, which was significantly lower than that of 1, 3 and 6 months [(29.27±9.07) μg/L, (27.79±10.18) μg/L and (31.98±12.55) μg/L respectively, all P 0.05). The BAC in efficacy group [(33.48±12.56) μg/L] was significantly higher than that in non-efficacy group [(21.46±6.00) μg/L] (t= 2.089, P= 0.035). 12.5% (5/40) of the patients had mild gastrointestinal side effects after treatment for 1 month, while the BAC of them [(16.93±1.80) μg/L] was significantly lower than that in patients without gastrointestinal side effects [(31.78±1.39) μg/L, P < 0.000 1]. The occurrence rate of abdominal pain and diarrhea was decreased after treatment for 3 and 6 months, while the BAC was increased gradually. Conclusions Compound QHP is effective in the treatment of MDS with mild adverse reactions. There is no damage to the heart, liver, and renal function. Besides, it shows that reducing the gastrointestinal adverse reactions and maintaining the effective concentration of BAC play a significant role in the effect of compound QHP in the treatment of MDS. Key words: Myelodysplastic syndromes; Arsenicals; Treatment outcome; Safety