Intravenous busulfan as preparative regimen in pediatric patients receiving hematopoietic stem cell transplantation: the preliminary experience in Taiwan.

BACKGROUND Some studies have proved that intravenous busulfan with cyclophosphamide (used as a component of conditioning regimens for hematopoietic stem cell transplantation) is safer and has fewer complication than oral busulfan in adults, whereas the same proof in pediatric patients is only limited, with no reported data so far from Asian countries. In this study, we aimed to evaluate the efficacy and complications of IV busulfan in pediatric patients. METHODS Three pediatric patients with acute myeloid leukemia were treated by intravenous busulfan combined with cyclophosphamide to compare retrospectively with the treatment with oral busulfan plus intravenous cyclophosphamide in another three pediatric cases having transplantation in the same institute. RESULTS The results showed that the intravenous busulfan-based regimen had better compliance and less adverse effects including mucositis, hepatic toxicity, transplant-related hepatic veno-occlusive disease, and acute graft-versus-host disease than oral busulfan-based treatment. CONCLUSIONS The conditioning regimen of intravenous busulfan combined with cyclophosphamide is an acceptable alternative for pediatric patients with hematological malignancies in Taiwan. The long-term benefit and adverse effects of intravenous busulfan should be further explored.