外周血造血干细胞移植治疗急性白血病50例疗效分析

Peripheral blood stem cell transplantation for patients with acute leukemia：clinical effects analysis in 50 cases

摘要:

目的：评价外周血造血干细胞移植（peripheral blood stem cell transplantation，PBSCT）治疗急性白血病（acute leukemia，AL）的临床疗效，比较异基因PBSCT（allogeneic PBSCT，Allo-PBSCT）与自体PBSCT（autologous PBSCT，Auto-PBSCT）在AL治疗方面的优缺点，探讨移植预处理方案的选择及疗效。方法：回顾性分析我院从2004-2012年间收治的50例因AL行PBSCT的病例，其中21例男性，29例女性，行Allo-PBSCT及Auto-PBSCT的分别为28例及22例。分析移植疗效及并发症，比较异基因及自体移植复发率、无病生存率（disease-free survival，DFS）及移植相关死亡率（transplant related mortality，TRM）的差异。分析预处理死亡及植入成功率。结果：50例患者中除2例MAC（马法兰+阿糖胞苷+环磷酰胺）方案预处理患者植入失败外，其余48例获得造血重建，移植成功率96%，中位白细胞植活时间（>0.5×109个/L）为11.3 d[（7~24） d]，中位血小板植活时间（＞20×109个/L）为14.3 d[（5~44） d]。异基因移植与自体移植相关死亡例数分别为8例、3例，TRM为28.6%、15.0%；累计DFS分别为46.4%、35.0%；累计复发率分别为25.0%、55.0%。随访时间内异基因及自体移植的总存活率（overall survival rate，OS）为57.1%、50.0%，两者移植前处于首次化疗缓解（complete remission at first course，CR1）及非首次化疗缓解或未缓解状态的患者，移植后OS分别为61.5%、53.3%及45.5%、55.6%；累计DFS分别为46.2%、46.7%及27.3%、44.4%；死亡率分别为38.5%、46.7%及54.5%、44.4%。结论：PBSCT是治疗AL的有效手段，特别是移植前状态处于CR1的患者移植效果更好。异基因移植后复发率较自体移植低，长期DFS较高，但移植相关并发症多，TRM较高。预处理方案的优化是提高移植疗效的关键，以马利兰/环磷酰胺方案为基础的预处理方案有较好的清髓效果及耐受。

Abstract:

Objective：To evaluate the therapeutic effect of peripheral blood stem cell transplantation（PBSCT） in the treatment of acute leukemia（AL），to discuss advantages and disadvantages of allogeneic PBSCT（Allo-PBSCT） and autologous PBSCT（Auto-PBSCT） and to choose the appropriate preparative transplantation regimen. Methods：All fifty patients with AL underwent PBSCT from 2004 to 2012 in our hospital were retrospectively analyzed including 21 male and 29 female. Twenty－eight patients were treated with Allo-PBSCT and 22 with Auto-PBSCT. Effects of transplantation and complications were analyzed. Differences in transplantation relapse rate，disease-free survival（DFS） and transplant related mortality（TRM） were compared. Preparative regimen related death and suc－cess rate were analyzed. Results：No pretreatment scheme related death was occurred except 2 cases of MAC（melphalan+Cytarabine+cyclophosphamide） preparative regimen implanted failure. Forty－eight patients engrafted successfully with transplant success rate of 96%. Median time of white blood cell>0.5×109/L was 11.3 d（（7-24） d） and platelets>20×109/L was 14.3 d（（5-44） d）. Allo-PBSCT and Auto-PBSCT related death cases were 8 cases and 3 cases respectively with TRM of 28.6% and 15.0%，respectively. During the follow-up，accumulative DFS for Allo-PBSCT and Auto-PBSCT were 46.4% and 35.0% respectively；accumulative relapse rates were 25.0% and 55.0%，respectively. During follow-up，overall survival（OS） rates of Allo-PBSCT and Auto-PBSCT were 57.1% and 50.0%，respectively. Among patients under the condition of complete remission at first course（CR1） and complete remission after two or more course or non-remission before transplantation，OS rates were 61.5%，53.3% and 45.5%，55.6%；DFS were 46.2%，46.7% and 27.3%，44.4%；death rates were 38.5%，46.7% and 54.5%，44.4%，respectively after transplantation. Conclusions：PBSCT is effective in the treatment of AL. Effect is better for patients under the condition of CR1 before transplantation. Relapse rate is lower，long-term DFS is higher and transplant related complications are more in Allo-PBSCT than in Auto-PBSCT. Optimization of preparative regimen is the key to improve transplantation efficacy，preparative regimen based on busulfan/cyclophosphamide regimen has a better effect of pulp and tolerance.