中文摘要：

背景：马利兰常用于骨髓或外周血干细胞移植预处理,其主要在肝脏代谢.目的：评价静脉应用马利兰预处理在造血干细胞移植治疗恶性血液病中的有效性及安全性.方法：纳入43例患者预处理方案采用静脉剂型马利兰,观察其造血干细胞移植预处理中马利兰治疗相关毒性（肝脏、神经系统、口腔黏膜炎）造血干细胞植活时间、急性移植物抗宿主病、总生存率、白血病复发情况.结果与结论：静脉应用马利兰后,谷丙转氨酶升高72%（31/43）,发生不典型肝静脉闭塞综合征5%（2/43）,有1例发生双手麻木.口腔黏膜炎发生率60%（26/43）.中性粒细胞植活时间16 d,血小板植活时间12 d,2例自体造血干细胞移植患者血小板延迟植活.Ⅰ~Ⅱ度急性移植物抗宿主病发生63%（24/38）.总生存率72%（31/43）.复发率23%（10/43）.提示造血干细胞移植预处理方案中应用静脉马利兰给药,患者耐受性好,方法安全有效.

英文摘要：

BACKGROUND： Busulfan is commonly used in bone marrow or peripheral blood stem cell transplantation pretreatment, which is mainly metabolized in the liver. OBJECTIVE： To evaluate the efficacy and safety of the conditioning regimen using intravenous busulfan in hematopoietic stem cell transplantation for malignant hematopathy. METHODS： A total of 43 patients receiving intravenous busulfan as conditioning regimen were included to observe regimen-related toxicity （hepatotoxicity, neurotoxicity, mucositis）, engraftment time, acute graft-versus-host disease （aGVHD）, overall survival, and leukemia relapse. RESULTS AND CONCLUSION： Thirty-one patients （72%） had a rise in the level of alanine aminotransferase. Untypical hepatic veno-occlusive syndrome occurred in 2 patients （5%）. One patient had numbness on a couple of hands. Twenty-six patients （60%） had mucositis. The engraftment time of neutrophil was 16 days and that of platelet was 12 days. Delayed platelet engraftment was in 2 cases of autologous hematopoietic stem cell transplantation. The occurrence of aGVHD Ⅰ-Ⅱ was 63% （24/38）. The overall survival was 72% （31/43）. The relapsed rate was 23% （10/43）. The conditioning regimen with intravenous busulfan in hematopoietic stem cell transplantation was effective and safe, and patients had better tolerance.