100例异体造血干细胞移植治疗恶性血液病移植前临床特征与预后相关性研究

Impact of pre-transplantation characteristics on the outcome of allogeneic hematopoietic stem cell transplantation in patients with hematological malignancies: a retrospective analysis of 100 cases

目的:分析异体造血干细胞移植(allo-HSCT)治疗恶性血液疾病移植前的临床特征与移植预后的关系。方法:100例接受allo-HSCT治疗患者中男63例,女37例,中位年龄34(15～55)岁。46例人类白细胞抗原(HLA)相合亲缘供体移植、51例HLA相合非亲缘无关供体和3例半相合亲缘供体移植。其中慢性粒细胞性白血病(CML)24例,急性髓细胞白血病(AML)37例,急性淋巴细胞性白血病(ALL)36例和骨髓增生异常综合征(MDS)3例。处于疾病早期患者78例,进展期患者22例。单因素和多因素方法分析患者年龄、性别、供患者HLA相合、干细胞来源(骨髓、外周血)、供受者性别、移植前疾病状态和诊断-移植时间等因素与移植后总生存(overall survival,OS)率、移植相关病死率(transplantation related mortality,TRM)和复发率(relapserate,RR)的关系。结果:100例患者随访24(1～84)个月,移植后5年预期OS率为(59.9±7.2)%,TRM为(24.9±6.5)%,RR为(22.4±5.9)%。单因素分析提示移植前疾病状态与移植后OS率、TRM、RR相关,诊断-移植时间与移植后OS率、TRM相关。多因素分析提示移植前疾病状态与OS率、RR相关,诊断-移植时间与TRM密切相关。结论:移植前疾病状态和诊断-移植时间对移植治疗的长期生存、复发和不良反应相关,早期移植是提高移植疗效关键。

Objective To assess the impact of pre-transplantation characteristics on the outcome of allogeneic hematopoietic stem cell transplantation （allo-HSCT） in patients with hematological malignancies. Methods The outcomes of 100 hematological malignancy patients received allo-HSCT （male 63, female 37; median age 34） were retrospectively analyzed. Forty six patients received allo-HSCT from human leucocyte antigen （HLA）-matched sibling donors and 54 from HLA-matched unrelated donors （n=51） or haploidentical donor （n=3）. Twenty-four patients were diagnosed as having chronic myeloid leukemia （CML） and 37 with acute myeloid leukemia （AML）, 36 with acute lymphocytic leukemia （ALL） and 3 with myelodysplasia syndrome （MDS）. Among these patients, 78 patients were transplanted with disease in early stage and 22 patients in advanced stage. Univariate and multivariate analysis were performed to identify the potential prognostic factors of allo-HSCT outcome in terms of overall survival （OS）, transplantation related mortality （TRM） and relapse rate （RR） which include age, sex, donor type, source of stem cell （bone marrow vs peripheral blood）, donor/recipient sex mismatch, pre- transplantation disease status and time from diagnosis to transplantation. Results The 100 patients were followed-up for a median of 24（1-84） mouths after transplantation with an estimated 5-year OS rate of （59.9±7.2）%, 5-year TRM of （24.9±6.5）% and 5-year RR of （22.4±5.9）%. Univariate analysis demonstrated that pre-transplantation disease status was correlated with OS, TRM and RR while the time from diagnosis to transplantation was correlated with OS and TRM. Multivariate analysis showed that disease status was the independent prognostic factor for both OS and RR while the time from diagnosis to transplantation was the independent prognostic factor for TRM. Conclusions Pre-transplantation disease status and time from diagnosis to transplantation were the prognostic factors for allo-HSCT in patients with hematological malignancies. Early transplantation is critical for achieving a good long-term outcome.