异基因造血干细胞移植治疗骨髓增生异常综合征6例报告

Allogeneic Hematopoietic Stem Cell Transplantation in 6 Patients with Myelodysplastic Syndrome

本研究探讨异基因造血干细胞移植(allo-HSCT)治疗骨髓增生异常综合征(MDS)的可行治疗方案及疗效。1999年9月-2007年2月采用清髓性预处理allo-HSCT治疗6例骨髓增生异常综合征患者,4例接受同胞HLA配型全相合的异基因外周血干细胞移植(allo-PBSCT),2例接受单倍体相合异基因骨髓移植(hi-allo BMT)。预处理清髓方案为:全相合移植采用Bu+Cy,单倍体相合异基因骨髓移植采用Cy+TBI+Ara-C。移植物抗宿主病(GVHD)采用联合免疫抑制剂的方法预防,全相合移植应用环胞菌素A和短程氨甲碟呤,单倍体相合异基骨髓移植除以上药物外还加用抗胸腺细胞球蛋白、CD25单克隆抗体及霉酚酸酯。结果表明,全部患者移植后均有造血重建,中性粒细胞数>0.5×109/L及血小板数>20×109/L的平均时间分别为15(11-20)天及20.3(12-28)天,植入证据检测证实为完全供者造血。仅1例发生急性1度皮肤移植物抗宿主病,其他患者未发生急性或慢性GVHD,1例移植后20个月死于疾病复发,1例移植后18个月死于肺部并发症,其余4例无病生存,中位存活时间51.8(18-108)个月。总之,allo-HSCT治疗MDS可行、有效,提高了年轻MDS患者疾病治愈及长期生存的机会。

This study was purposed to explore the efficacy of hematopoietic reconstitution and survival of patients with myelodysplastic syndrome （MDS） after allogeneic hematopoietic stem cell transplantation （allo-HSCT）. Allo- HSCT without T lymphocyte depletion was used in 6 patients with MDS from November 1999 to June 2007. 4 cases out of them received allo-PBSCT from HLA matched sibling donors with conditioning regimen of cyclophosphamide （CTX） and Bu. Graft versus host disease（GVHD） was prevented by the administration of immunosuppressive drugs of cyclospofine A（CsA） and short-course MTX. 2 patients received haploidentical allogeneic bone marrow transplantation （hi- alloBMT） after preconditioning with cytosine arabinoside （Ara-C）, CTX and total body irradiation （TBI） with a linear accelerator. GVHD was prevented by the administration of immunosuppressive drugs including CSA, short-course MTX, MMF, anti-CD25 monoclonal antibody and ATG. The results showed that all of the patients were engrafted successfully. The median time of granulocyte recovery exceeding 0.5 ×10^9/L and platelets exceeding 20 ×10^9/L were days 15 and 20.3 respectively, and 100% donor hematological ceils were detected by cytogenetic analysis. All patients did not expe- rience serious acute graft-versus-host disease （aGVHD）. During 18 -108 months of following-up, 2 cases died of pulmonary complication and of relapse ; the other 4 cases survive in a disease-free situation. In conclusion, allo-HSCT was an effective approach for the treatment of MDS.