非血缘脐血移植与HLA相合同胞外周血干细胞移植治疗成人恶性血液病的对比研究

Comparison of unrelated cord blood transplantation and HLA-identical sibling peripheral blood stem cell transplantation for the treatment of adult hematological malignancies

目的比较单份清髓非血缘脐血移植（UCBT）与HLA相合同胞外周血干细胞移植（PBSCT）治疗成人恶性血液病的疗效。方法自2011年4月1日至2015年12月31日，共81例单份清髓UCBT与57例HLA相合同胞PBSCT患者人组进行比较。所有患者均采用清髓性预处理方案，环孢素A联合霉酚酸酯预防移植物抗宿主病（GvHD）。结果移植后42d（＋42d）ucBT组中性粒细胞累积植入率为95．0％（95％C187．0％～98．1％），同胞PBSCT组为100％（P=0．863）。＋100dUCBT组血小板累积植入率低于同胞PBSCT组[87．3％（95％CI76．8％～93．1％）对98．2％（95％CI87．3％～99．7％），P=0．005]。＋100dUCBT组Ⅱ-Ⅳ度急性GVHD、Ⅲ／Ⅳ度急性GVHD的累积发生率分别为18．2％（95％a9．1％-26．4％）、10．4％（95％CI3．3％～16．9％），同胞PBSCT组分别为8．9％（95％C11．1％～16．1％）、7．1％（95％C11．4％～13．7％），差异均无统计学意义（P=0．142，P=0．521）。UCBT组3年慢性GVHD、广泛型慢性GVHD累积发生率均低于同胞PBSCT组[14．9％（95％CI5．2％～23．5％）对35．2％（95％CI19．4％～47．8％），P=0．008；11．2％（95％c，2．9％～18．7％）对31．4％（95％a16．2％～43．9％），P=0．009]。UCBT组、同胞PBSCT组3年移植相关死亡率（TRM）差异无统计学意义[30．1％（95％CI19．2％-41．7％）对23．2％（95％C113．1％～35．0％），P=0．464]。UCBT组3年疾病复发率低于同胞PBSCT组[12．9％（95％凹6．6％-21．5％）对24．3％（95％CI13．5％～36．8％），P=0．039]。UCBT、同胞PBSCT组3年总生存率、无病生存率差异均无统计学意义[58．6％（95％C145．4％～69．7％）对54．8％（95％C139．9％-67．4％），P=-0．634；57．0％（95％C144．0％～68．0％）对52．4％（95％CI38．2％～64．9％），P=0．563]。UCBT组3年无GvHD及复发生存（GRFS）率高于同胞PBSCT组[55．7％（95％C144．1％～65．8％）对42．9％（95％CI29．8％-55．3％），P=0．047]。结论对于成人恶性血液病，UCBT与HLA相合同胞PBSCT具有相似的急性GVHD发生率、TRM、总生存率和无病生存率，但UCBT组具有较低的慢性GVHD发生率及疾病复发率。

Objective To compare the efficacy of unrelated cord blood transplantation （UCBT） and HLA-identical sibling peripheral blood stem cell transplantation （PBSCT） for the treatment of adult hematological malignancies. Methods From April 2011 to December 2015, a total of 81 patients receiving single-unit UCBT and 57 patients receiving HLA-identical sibling PBSCT were enrolled in thisstudy. All of the patients received myelablative conditioning. Cyclosporine combined with mycophenolate mofetil was adopted for GVHD prophylaxis. Results The cumulative incidence of neutropil engraftment at day-42 was 95.0% and 100% in UCBT and sibling PBSCT groups, respectively （P=0.863）. Platelet engraftment at day 100 was 87.3% （95%CI 76.8%-93.1%） in UCBT group, which was significantly lower than that of sibling PBSCT group [98.2% （95%CI 87.3%-99.7%） ] （P=0.005）. There were no significant differences in terms of 11 -IV acute GVHD or llI- IV acute GVHD in two groups （P=-0.142, 0.521 ）. The 3- year chronic GVHD and extensive chronic GVHD were 14.9% （95%CI 5.2%-23.5%） and 11.2% （95%CI 2.9%-18.7% ）, respectively in UCBT group, which was significantly lower than that of sibling PBSCT group [35.2% （95%CI 19.4%-47.8% ）, 31.4% （95%CI 16.2%-43.9% ） （P=-0.008, 0.009）. The 3-year transplant-related mortality （TRM） was similar between two groups （30.1% vs 23.2%, P=0.464）. The relapse rate at 3-year in UCBT group [ 12.9% （95%CI 6.6%-21.5%） was significantly lower than that in sibling PBSCT group I24.3%（95%CI 13.5%-36.8%） （P=0.039）. There were no significant differences in terms of overall survival （OS） and disease-free survival （DFS） between two groups （58.6% vs 54.8%, P=-0.634; 57.0% vs 52.4%, P=-0.563）. But GVHD-free and relapse-free survival （GRFS） in UCBT group [ 55.7% （95% CI 44.1%- 65.8% ） ] was significantly higher than that of sibling PBSCT group 142.9% （95% CI 29.8%-55.3% ） （P=-0.047）. Conclusions For adult hematological malignancies, the incidences of acute GVHD and TRM were similar between UCBT and sibling PBSCT recipients, and the incidences of chronic GVHD and relapse were lower in UCBT recipients. UCBT recipients had higher GRFS rate although OS and DFS were similar between two groups, which may reflect the real recovery and better quality of life following UCBT.