OBJECTIVE:To evaluate the relationship of blood concentration of busulfan(Bu) with treatment outcome of hematopoietic stem cell transplantation(HSCT).METHODS:Therapeutic efficacy and toxicity were observed in 22 HSCT patients who pre-treated with intravenous dosage form of Bu.Blood concentration of Bu was monitored by pre-column derivatization HPLC before and after medication,and AUC of Bu was calculated.RESULTS:The probabilities of developing gastrointestinal toxicity,hepatotoxicity,mucositis and GVHD increased with increases of AUC.Further,the risk of death was significantly lower for patients having an AUC0~6 h between approximately 900 and 1 400 μmol·min·L-1,whereas the risk increased sharply with either lower or higher AUC values.CONCLUSION:Drug dosage is adjusted timely according to blood concentration monitoring of Bu,and it improves clinical prognosis of HSCT patients and reduction of post-operative complication.