异体造血干细胞移植治疗白血病的长期疗效和预后分析

Long-term outcome and prognosis in leukemia patients treated with allogeneic hematopoietic stem cell transplantation

目的分析异体造血干细胞移植(Allo-HSCT)治疗白血病的长期疗效和相关预后因素。方法1999年4月～2007年12月,应用Allo-HSCT治疗各类白血病患者37例,其中第1次完全缓解期急性髓细胞白血病(AML)和急性淋巴细胞白血病(ALL)各11例,慢性期的慢性粒细胞性白血病(CML)15例。在37例中,HLA全相合同胞供体外周血造血干细胞移植20例,HLA相合非血缘供体造血干细胞移植17例(骨髓14例,外周血3例);移植预处理包括环磷酰胺(Cy)+全身照射(TBI)±依托泊苷(VP- 16)方案21例、白消安(Bu)+Cy方案11例和氟达拉宾(Flud)+阿糖胞苷(Ara-C)+Bu方案5例;异基因移植患者预防移植物抗宿主病(GVHD)方案为氨甲碟啶(MTX)+环孢素(CSP)±吗替麦考酚酯(MMF)。对37例患者的总体生存率、移植相关死亡率(TRM)、复发率进行统计,并分析相关预后因素。结果移植后预期6年总体生存率为(67.6±10.1)%;移植后第100天TRM为(2.7±2.7)%,预期6年总体TRM为(28.6±9.9)%;预期6年复发率为(5.3±5.5)%。单因素和多因素分析提示,发生重度GVHD(Ⅱ～Ⅳ度急性GVHD或广泛性慢性GVHD)是影响生存的最重要因素。结论Allo-HSCT治疗白血病具有良好的长期疗效。预防和治疗GVHD对进一步提高Allo-HSCT的长期疗效和生存率具有重要意义。

Objective To evaluate the long-term outcome of leukemia patients treated with allogeneic hematopoietic stem cell transplantation （Allo-HSCT） and the potential prognostic factors. Methods A total of 37 leukemia patients treated with Allo-HSCT were included in the analysis, 11 with acute myeloid leukemia （AML） in first remission, 11 with acute lymphocytic leukemia （ALL） in CR1 and the other 15 with chronic myelocytie leukemia （CML） in chronic phase. All patients received either HLA comparable sibling mobilized peripheral blood hematopoietie stem cells （n = 20） or HLA identical bone marrow （n = 14） or mobilized peripheral blood （n = 3） from unrelated donors. The preconditioning regimens were standard TBI containing regimen （n =21, Cy ＋ TBI ＋_ VP-16） and chemotherapy regimen （ Bu ＋ Cy, n = 11 and Flud ＋ Ara-C ＋ Bu, n = 5）. All patients received standard graft-verse-host disease（GVHD） prophylaxis with MTX ＋ CSP ＋MMF. All patients were followed up and evaluated for survival, transplantation-related mortality （TRM） , relapse rate and prognostic factors. Results The predicted 6-year overall survival was （67.6 ± 10.1 ） % , with 100-d TRM of （2.7 ± 2.7） % and 6-year TRM of （28.6 ± 9.9） % , respectively. The predicted 6-year relapse rate was （5.3 ± 5.5） %. It was demonstrated by univarite and multivariate analysis that the occurrence of severe GVHD （Ⅱ-Ⅳ acute GVHD/extensive chronic GVHD） was the most important pronsositc factor. Conclusion Allo-HSCT yields a satisfactory long-term outcome in leukemia patients. Progress in GVHD prophylaxis and/or treatment is critical for the further improvement of Allo-HSCT outcome.