Intravenous busulfan for 83 pediatric patients undergoing hematopoietic stem cell transplantation: A single center experience

Abstract

The intravenous form (IV) of busulfan (Bu) can be stably administered, but there are few large-scale studies on its safety and efficacy in children. We performed a retrospective analysis of 83 pediatric patients who received IV Bu before undergoing stem cell transplantation at our center from January 2007 to September 2017. In these 83 patients (44 with solid tumors, 13 with acute myeloid leukemia, 12 with acute lymphocytic leukemia, 6 with myelodysplastic syndrome, 2 with malignant lymphoma, 2 with hematopoietic disorder, 2 with immunodeficiency, and 2 with others), stem cell transplantation was performed 90 times, including 77 instances of myeloablative conditioning. The median age was 5 years (range, 5 months–16 years), and the male:female ratio was 50:33. We performed autologous hematopoietic stem cell transplantation in 50 patients and allogeneic hematopoietic stem cell transplantation in 40 patients. The three-year overall survival (54.0% vs 59.1%, p=0.34), 1-year nonrelapse mortality (18.7% vs 21.4%, p=0.39), and engraftment rates (88.2% vs 96.7%, p=0.65) did not significantly differ between the IV-BU- and oral-BU-treated groups. Twenty-seven (30%) patients received Bu once daily. Three patients showed interstitial pneumonia (3.3%), 13 showed hepatic sinus occlusion syndrome (14.4%), 4 showed central nervous complications (4.4%), and 6 showed bronchiolitis obliterans (6.7%), but there were no significant differences between the IV-BU-treated and oral-BU-treated groups. The survival rates and adverse effects were similar in both the once-daily and four-times-daily IV-BU-treated groups. In conclusion, the efficacy and safety of IV Bu in pediatric patients are equivalent to those of oral Bu. Bu administration once daily exhibited similar safety and efficacy.