异基因造血干细胞移植治疗骨髓增生异常综合征30例分析

Allogeneic stem cell transplantation from genotypically HLA-identical siblings for 30 patients with myelodysplastic syndromes

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 explore the indication and optimum time for treating myelodysplastic syndrome （MDS） by allogeneic hematopoietic stem cell transplantation（allo-HSCT） with HLA identical sibling grafts. Methods From June 1997 to Sep. 2004, a total of 30 patients with MDS were treated with allo-HSCT from HLA-identical sibling donors in our institute. On HSCT, 4 patients had refractory anemia （RA） , 2 RA with ringed sideroblasts （RARS） , 7 RA with excess blasts（ RAEB ）, 14 RAEB in transformation （RAEB- t） , 3 already progressed to secondary AML. For IPSS system, 6 patients were in intermediate- Ⅰ risk group, 11 in intermediate-Ⅱ risk group, and 13 in high risk group. The modified BU/CY conditioning regimen was used. Four patients received bone marrow transplantation（BMT） , 8 received peripheral blood stem cell trans- plantation（ PBSCT）, and 18 received BMT ＋ PBSCT. Results The 3-year expected overall survival （OS） was 63.61% , 3-year expected disease-free survival （DFS） 61.41% , and relapse rate 5.26% ; OS for RA/ RAS, RAEB and RAEB-t/AML subgroup was 83.33% ,34.29% and 66.67% , respectively, and all had no statistic difference among them. OS for IPSS-intermediate and high risk subgroup was 64.7% , and 69.0% respectively, also had no statistic difference. 3-year expected OS in no aGVHD ,grade Ⅰ-Ⅱ aGVHD and grade Ⅲ-Ⅳ aGVHD group was 57.75% , 100% and 0% , respectively（P = 0. 009）. Pre-HSCT chemotherapy, disease subtype and cGVHD all had no correlation with LFS or OS （ P 〉 0.05 ）. Conclusion For young MDS patients having HLA-identical sibling donors, HSCT should be the first line therapy and performed as soon as possible.