异基因外周血干细胞移植治疗骨髓增生异常综合征8例临床分析

Clinical analysis of allogeneic peripheral blood stem cell transplantation on treating myelodysplastic syndrome in 8 cases

目的观察异基因外周血干细胞移植（allogeneic peripheral blood stem cell transplantation，allo—PBSCT）治疗骨髓增生异常综合征（myelodysplastic syndrome，MDS）的疗效。方法对2007年5月至2012年1月在中山大学附属第三医院血液科住院的8例MDS患者采用allo—PBSCT治疗。其中男2例，女6例，年龄18～50岁（中位年龄31岁）。按世界卫生组织（Wodd Health Organization，WHO）MDS分型标准（2008年）分类，难治性细胞减少伴多系增生异常（refractory cytopenia with multilineage dysplasia，RCMD）占2例，难治性贫血伴原始细胞增多（refractory anemia with excess blasts，RAEB）-1占1例，RAEB-2占3例，MDS进展为急性髓系白血病（MDS-AML）占2例。供者经粒细胞集落刺激因子（granulocyte colony-stimulating factor，G—CSF）5～10μg／（kg·d）动员5d后，分1～2d采集外周血造血干细胞。受者输入外周血单个核细胞（5．8—11．6）×10^8／kg（中位数7．7×10^8／kg）。受者预处理方案为BuCy方案6例，改良BuCy方案2例，非亲缘供者联合使用抗胸腺细胞球蛋白（ATG）。予环孢素加短疗程甲氨蝶呤方案预防移植物抗宿主病（GVHD）。结果8例患者中有6例移植后造血重建，中性粒细胞≥0．5×10^9／L和血小板≥20×10^9／L的时间分别为移植后9—15d（中位数12d）及11～42d（中位数13d）；4例患者发生急性移植物抗宿主病（aGVHD），其中Ⅰ度1例，Ⅱ度2例，Ⅳ度1例；存活超过100d的6例患者发生局限型慢性移植物抗宿主病3例。随访时间为2．6—56．9个月（中位数27．0个月），2例患者移植后100d内死亡，其余患者均无病存活。以Kaplan—Meier法估算，患者总体生存率及无病生存率均为（75．0±15．3）％，生存期为（43．4±8．3）个月。结论外周血干细胞移植是治疗年轻MDS患者的有效方.

Objective To observe the therapeutic effect of a11ogeneic peripheral blood stem cell transplantation （allo-PBSCT） on myelodysplastic syndrome （MDS）. Methods From May 2007 to January 2012, 8 patients with MDS were treated with allo-PBSCT in Department of Hematology of the Third Affiliated Hospital of Sun Yat-sen University, including 2 males and 6 females with the age ranged from 18 to 50 years old （median age 31 years old）. According to MDS classification standard （2008） from World Health Organization （WHO）, patients were classified as refractory cytopenia with muhilineage dysplasia （RCMD, n = 2）, refractory anemia with excess blasts-1 （ RAEB-1, n = 1 ）, RAEB-2 （n = 3） and MDS progressed to acute myeloid leukemia （MDS-AML, n = 2）. Hematopoietie stem eells were mobilized from the donor＇s peripheral blood after administering granulocyte-colony stimulating factor （G-CSF） at a dose of 5-10 μg / （ kg · d） for 5 days, with apheresis for 1 or 2 days. Peripheral mononucleated cells were given to recipients with a dose of （5.8-11.6） × 10^8/kg （median 7.7 × 10^8/kg）. The BuCy conditioning regimen was used in 6 cases, and the modified BuCy conditioning regimen was used in 2 cases, and antithymocyte globulin （ATG） was jointly used for unrelated PBSCT. Ciclosporin and methotrexate for short-course were used to prevent graft-versus-host disease （GVHD）. Results The haematopoietic system were successfully reconstructed in 6 cases. The time of granulocyte recovery ≥ 0. 5 × 10^9/L and platelets ≥ 20 × 10^9/L was 9-15 d （median 12 d） and 11- 42 d （median 13 d） respectively. Four eases developed acute GVHD （aGVHD） （1 case of grade I , 2 eases of grade 11 , 1 case of grade 1V ）. Six eases survived for more than 100 d and 3 of them developed chronic GVHD （eGVHD）. Follow-up time was 2. 6-56. 9 mon（ median 27.0 mon）. Two cases died in 100 d after PBSCT, and the other 6 eases survived with disease-free. By Kaplan-Meier method, overall survival （OS） and disease-free survival （DFS） rates were both （75.0 ± 15.3 ）% , and estimated mean survival time was （43.4 ± 8.3 ） mon. Conclusions Allo-PBSCT is an effective treatment for young MDS patients.