Abstract:

BACKGROUND: Erratic gastrointestinal absorption as a result of oral administration of busulfan not only affects the efficacy, but also increases the risk of toxicity. There are few papers about intravenous busulfan in conditioning regimen for unrelated allogeneic hematopoietic stem cell transplantation although it is used in China for several years.
OBJECTIVE: To study the efficacy and associated complications of intravenous busulfan in conditioning regimens for unrelated allogeneic hematopoietic stem cell transplantation.
METHODS: Fourteen patients received intravenous busulfan + cyclophosphamide before unrelated allogeneic hematopoietic stem cell transplantation, while 18 patients received oral busulfan + cyclophosphamide before related transplantation. The therapeutic indices and the adverse events including gastrointestinal tract reaction, oral mucositis, hemorrhagic cystitis, hepatic function lesion, acute graft versus-host disease in two groups were assessed.
RESULTS AND CONCLUSION: All patients had hematopoietic engraftment. The hepatic damage and oral mucositis in the intravenous busulfan group were significantly lower than those in the oral busulfan group (14% vs. 67%, 7% vs. 55%, P < 0.01). There were no significant differences in gastrointestinal tract reaction, hemorrhagic cystitis, reconstruction, acute graft-versus-host disease, and chronic-versus-host disease between two groups. The intravenous busulfan combined with cyclophosphamide in the treatment of unrelated allogeneic hematopoietic stem cell transplantation acquires satisfactory efficacy with low toxicity.