白消安在非亲缘异基因造血干细胞移植预处理中的应用  

Busulfan for unrelated allogeneic hematopoietic stem cell transplantation

作　　者：李旭东[1,2] 何易[1,2] 王东宁[1,2] 陆英[1,2] 林东军[1,2] 肖若芝[1,2] 黄仁魏[1,2] 

机构地区：[1]中山大学附属第三医院血液科 [2]中山大学血液病研究所,广东省广州市510630

出　　处：《中国组织工程研究》2012年第45期8460-8464,共5页Chinese Journal of Tissue Engineering Research

摘　　要：背景:口服白消安剂型胃肠道吸收不稳定,影响异基因造血干细胞疗效且毒性增加。静脉剂型白消安在国内最近几年用于临床,但在非亲缘异基因移植预处理中应用的相关报道甚少。目的:探讨静脉剂型白消安在非亲缘异基因造血干细胞移植预处理中应用的疗效并观察其毒副作用。方法:14例非亲缘异基因干细胞移植采用静脉剂型白消安联合环磷酰胺预处理方案,18例亲缘异基因干细胞移植采用口服白消安联合环磷酰胺预处理方案,观察两组造血重建、植入率等疗效指标及胃肠道反应、口腔黏膜炎、出血性膀胱炎、肝功能损害、移植物抗宿主病等相关毒性指标。结果与结论:两组患者植入率均为100%。静脉剂型组肝脏毒性、口腔黏膜炎发生率明显低于口服剂型组(14%vs.67%,7%vs.55%),差异均有显著性意义(P<0.01),而胃肠道反应、出血性膀胱炎、造血重建、急性移植物抗宿主病、局限性移植物抗宿主病等方面差异均无显著性意义(P>0.05)。结果可见静脉剂型白消安应用于非亲缘异基因造血干细胞移植预处理可获得满意疗效,且毒副反应少。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.

关 键 词：造血干细胞移植 非亲缘供者 静脉剂型 白消安 造血重建 不良反应 

分 类 号：R394.2[医药卫生—医学遗传学]