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目的探讨同胞配型相合的异基因造血干细胞移植(allo-HSCT)治疗骨髓增生异常综合征(MDS)的适应证和移植时机。方法 1997年6月至2004年9月采用同胞相合 allo-HSCT 治疗 MDS30例。预处理主要采用改良 BU/CY 方案,4例接受骨髓移植(BMT),8例接受外周血造血干细胞移植(PBSCT),18例接受 BMT 联合 PBSCT。结果 3年预期存活(OS)率63.61%,3年预期无病存活(DFS)率61.41%,复发率(RI)5.26%。OS 率在 RA/RAS 组83.33%,RAEB 组34.29%,RAEB-t/AML组66.67%,组间比较差异无统计学意义(P=0.563);以 IPSS 分组显示3年预期存活率:中危组64.7%,高危组69.0%,组间比较差异无统计学意义(P>0.05)。单因素分析发现,无急性移植物抗宿主病(GVHD)组、Ⅰ～Ⅲ度 GVHD 组和Ⅲ～Ⅳ度 GVHD 组 OS 率分别为57.75%,100% 和0%,组间比较差异有统计学意义(P=0.009);而患者移植前是否治疗、疾病分期、慢性 GVHD 均不是 DFS 率的影响因素(P>0.05)。结论如有 HLA 相合的同胞供者,HSCT 可作为年轻的中高危 MDS 患者的一线治疗,建议采用 BU/CY 方案和 PBSCT。MDS 的最佳治疗策略尚须要进一步进行大规模随机对照研究。
Objective To investigate the indications and the timing of transplantation of allo-HSCT in the treatment of myelodysplastic syndrome (MDS). Methods From June 1997 to September 2004, 30 cases of MDS were treated with sibling allo-HSCT. Pretreatment consisted mainly of modified BU / CY regimen, 4 patients received BMT, 8 received PBSCT and 18 received BMT combined with PBSCT. Results The expected OS rate was 63.61% at 3 years, 61.41% at DFS and 5.26% at 3 years. OS rates were 83.33% in RA / RAS group, 34.29% in RAEB group and 66.67% in RAEB-t / AML group, with no significant difference between the two groups (P = 0.563). The 3-year expected survival rate 64.7% in the group and 69.0% in the high risk group. There was no significant difference between the two groups (P> 0.05). Univariate analysis showed that OS rates were 57.75%, 100% and 0% in GVHD group, grade Ⅰ ~ Ⅲ GVHD group and grade Ⅲ ~ Ⅳ GVHD group, respectively, with statistical difference between the two groups (P = 0.009). However, whether patients were treated before transplantation, stage of disease or chronic GVHD were not the factors influencing DFS rate (P> 0.05). Conclusions HSCT can be used as a first-line treatment for young, middle-high-risk MDS patients with HLA-matched sibling donors. BU / CY regimen and PBSCT are recommended. The best treatment strategy for MDS still requires further large-scale randomized controlled studies.