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目的评价造血干细胞移植(HSCT)治疗骨髓增生异常综合征(MDS)患者的疗效,探讨MDS患者接受HSCT治疗的适应证和时机。方法1993年11月～2007年4月对20例MDS及MDS转急性髓系白血病(AML)患者(男性12例,女性8例,中位年龄39岁)行HSCT治疗。其中18例接受同胞供者异基因外周血干细胞移植(allo-PBSCT);1例为同基因骨髓移植(Syn-BMT);1例为无关脐带血移植(CBT),+25d时移植失败行自体骨髓移植。预处理主要采用修改的Bu/Cy方案。结果3年总生存率(OS)及3年无病生存率均为53.3%±12%;3年复发率(RR)10.8%±7%,移植相关死亡率(TRM)42.6%±12%。截止随访日期,存活11例,中位生存时间16.5(2.0～112)个月。结论HSCT是治疗MDS的有效方法,如有HLA匹配的同胞供者,HSCT可作为MDS患者的一线治疗。
Objective To evaluate the efficacy of hematopoietic stem cell transplantation (HSCT) in the treatment of patients with myelodysplastic syndrome (MDS) and to explore the indications and timing of HSCT in patients with MDS. Methods From November 1993 to April 2007, 20 patients with MDS and MDS with acute myeloid leukemia (AML) (12 males and 8 females, with a median age of 39 years) underwent HSCT. 18 cases received sibling donor allogeneic peripheral blood stem cell transplantation (allo-PBSCT); 1 case was syngeneic bone marrow transplantation (Syn-BMT); 1 case had unrelated cord blood transplantation (CBT) Bone marrow transplantation. Pretreatment mainly uses the modified Bu / Cy scheme. Results The 3-year OS and 3-year disease-free survival rates were 53.3% ± 12%. The 3-year relapse rate was 10.8% ± 7% and the transplant-related mortality rate was 42.6% ± 12%. As of the follow-up date, 11 patients survived, and the median survival time was 16.5 (2.0-112) months. Conclusion HSCT is an effective method for the treatment of MDS. HSCT can be used as a first-line treatment for patients with MDS if HLA matched sibling donors.