自体或异基因外周血造血干细胞移植治疗恶性血液病53例(英文)

Autologous or allogeneic peripheral blood stem cell transplantation for the malignant hematologic diseases in 53 cases

背景：造血干细胞移植使恶性血液病的预后得到很大的改观,外周血造血干细胞移植逐渐取代了骨髓移植而成为造血干细胞移植的主要方式。目的：观察自体或异基因外周血造血干细胞移植对53例恶性血液病患者的治疗效果。设计：随机对照观察。单位：郑州大学第一附属医院血液科骨髓移植中心。对象：选择2003-07/2006-05在郑州大学第一附属医院血液科收治的53例恶性血液病患者,男33例,女20例,平均37岁。35例患者行异基因外周血造血干细胞移植,其中急性髓细胞性白血病13例、急性淋巴细胞白血病7例、慢性粒细胞白血病10例、多发性骨髓瘤2例、骨髓增生异常综合征3例。18例患者行自体外周血造血干细胞移植,其中急性髓细胞性白血病7例、急性淋巴细胞白血病6例、多发性骨髓瘤2例、非霍奇金病3例。33名供者与受者HLA配型完全相合,2名1个位点不合,男20例,女13例,平均35岁。性别不相合者16例。实验经过医院伦理委员会批准许可,所有受试对象均对检测项目知情同意。方法：①利用粒系集落刺激因子或化疗联合粒系集落刺激因子动员外周血干细胞,自体移植患者接受CD34＋细胞中位数为3.0×10^6/kg,异基因移植患者接受CD34＋细胞中位数为6.2×10^6/kg;自体移植采用MAC预处理方案,异基因移植采用改良的Bu/Cy预处理方案;移植物抗宿主病的预防采用甲氨喋呤、环孢菌素A联合骁悉,1例1位点不合患者加用抗淋巴细胞球蛋白。②观察患者移植后中性粒细胞〉0.5×10^9L^-1和血小板〉20×10^9L^-1恢复时间。③观察患者移植物抗宿主病发生情况。④术后1年随访所有患者复发情况。主要观察指标：①中性粒细胞和血小板恢复时间。②移植后移植物抗宿主病发生情况。③随访结果。结果：纳入患者53例均进入结果分析。①自体移植患者移植后中性粒细胞〉0.5×10^9L^-1、血小板〉20×10^9L^-1的恢复时间分别为13和19d,在异基因移植患者中分别为12和15d。②异基因移植患者中Ⅰ-Ⅲ度急性移植物抗宿主病的发生率为31.4%,慢性移植物抗宿主病的发生率为71.4%。③术后患者自体移植和异基因移植患者复发率分别为38.9%和5.7%。结论：自体或异基因外周血造血干细胞移植较快建立恶性血液病患者造血能力,是治疗恶性血液病的重要手段。

BACKGROUND： The prognosis of malignant hematologic diseases has improved greatly with the application of the hematopoietic stem cell transplantation. Peripheral blood stem cell transplantation （PBSCT） has been used as an alternative to bone marrow transplantation （BMT）. OBJECTIVE： To observe curative effect and clinical outcome in 53 patients with hematological malignancy undergoing allogeneic peripheral blood stem cell transplantation （allo-PBSCT） or autologous peripheral blood stem cell transplantation （auto-PBSCT）. DESIGN： Randomized controlled study. SETTING： BMT Center, Hematology Department of the First Affiliated Hospital of Zhengzhou University. PARTICIPANTS： From July 2003 to May 2006, 53 patients （33 males and 20 females） with a median age of 37 years underwent PBSCT. Thirty-five patients received allo-PBSCT, including 13 of acute myelocytic leukemia （AML）, 7 of acute lymphocytic leukemia （ALL）, 10 of chronic myelocytic leukemia （CML）, 2 of multiple myeloma （MM）, and 3 of myelodysplastic syndrome （MDS）. Eighteen patients underwent auto-PBSCT, including 7 AML, 6 ALL, 2 MM, and 3 non-Hodgkin lymphoma （NHL）. Thirty-three donors （20 males and 13 females） with a median age of 35 age in the aIIo-PBSCT were HLA-identical siblings, 2 donors （5.7%） had one mismatch. Sixteen allografts were sex mismatched. Study was authorized by the Ethic committee of the hospital, and all patients had signed an inform consent. METHODS： （1） PBSC were mobilized with granulocyte colony-stimulating factor （G-CSF） or chemotherapy combined with G-CSF. A median of 6.2×10^6 CD34^＋ cells/kg was infused for allo-PBSCT and ×10^6 CD34^＋ cells/kg was infused for auto-PBSCT Amended BU/CY was used as conditioning regimen in aIIo-PBSCT and MAC was used in auto-PBSCT. Methotrexate （MTX） combined with cyclosporine A （CsA） and mycophenolate mofetil （MMF） was used as graft-versus-host disease （GVHD） prophylaxis. ALG was used in l patient with l locus mismatched in aIIo-PBSCT. （2） Time to engraftment was calculated from the time of transplantation to neutrophil recovery ≥ 0.5×10^9 ·L^-1 and platelet recovery ≥ 20×10^9 ·L^-1 , GVHD and relapse were observed until 1 year of follow-up. MAIN OUTCOME MEASURES： （1） Time to neutrophil and platelet recovery; （2） GVHD occurrence after transplantation; （3） outcome of treatment. RESULTS： All the 53 patients were analyzed. （1） Engraftment of neutrophils （〉 0.5×10^9 ·L^-1） and platelets （〉 20×10^9 ·L^-1） was achieved at a median of 13 days for neutrophils and 19 days for platelets in auto-PBSCT, and 12 days and 15 days respectively in alIo-PBSCT. （2） In allo-PBSCT, I-VI acute GVHD occurred in 31.4% cases, and chronic GVHD developed in 71.4% cases. （3） The relapse rate was 38.9% in auto-PBSCT, and 5.7% in alIo-PBSCT. CONCLUSION： PBSCT can provide rapid hematopoietic reconstitution. It is an important method to cure the malignant hematologic diseases.