摘要
目的 探讨同胞全相合异基因造血干细胞移植(allo-HSCT)治疗骨髓增生异常综合征(MDS)的疗效与时机.方法 回顾分析2003年1月-2012年12月采用同胞全相合allo-HSCT治疗MDS及MDS转急性髓性白血病(AML) 95例.采用改良马利兰+环磷酰胺或氟达拉滨的预处理方案,行骨髓和/或外周血干细胞移植.结果 95例患者中93例白细胞植活,Ⅱ~Ⅳ度急性移植物抗宿主病(aGVHD)累计发生率为12.9%±3.5%;慢性移植物抗宿主病(cGVHD)3年累计发生率为80.3%±4.9%.3年累计复发率(RR)为25.9%±4.7%,非复发死亡率(NRM)为16.1%±4.0%.3年预期总生存(0S)率及无病生存(DFS)率分别为69.9%±5.0%和58.0%±5.4%.多因素分析显示,发生Ⅱ~Ⅳ度aGVHD和不发生cGVHD是OS的独立危险因素;国际预后积分系统(IPSS)分组是DFS的独立预后因素.将难治性贫血伴原始细胞增多转化型(RAEB-t)及MDS转AML患者(31例)分为移植前未化疗、化疗未缓解、化疗缓解3组,3年OS率分别为33.9%、32.7%、100.0%,化疗缓解组OS率明显高于另外两组(P<0.05),DFS率、RR率差异无统计学意义.结论 同胞全相合allo-HSCT是治疗MDS的有效手段,IPSS可预测移植后疗效,对于移植前疾病进展的患者,争取缓解后行allo-HSCT可能提高疗效,但尚需进一步临床对照研究.
Objective To evaluate the efficacy and optimize the timing of allogeneic hematopoietic stem cell transplantation (allo-HSCT) from human leukocyte antigen (HLA)-identical siblings for myelodysplastic syndrome (MDS). Methods From January 2003 to December 2012, 95 patients with MDS or secondary acute myeloid leukemia (AML) were treated with HLA-identical allo-HSCT in our hospital. The median age was 43 (21-59) years. Conditioning regimens including modified busulfan (Bu)/ cyclophosphamide (Cy) or Bu/fludarabine (Flu) were used. All patients received transfusion of donor stem cells mobilized by granulocyte colony-stimulating factor (G-CSF) from bone marrow and/or peripheral blood. Eleven patients had refractory anemia (RA) or RA with ringed sideroblasts, 53 of RA with excess blasts (RAEB) , 15 of RAEB in transformation ( RAEB-t), and 16 progressing to secondary AML. Results A total of 93 patients achieved sustained myeloid engraftment. The cumulative incidence of grade Ⅱ-Ⅳ acute graft versus host disease (aGVHD) was 12. 9% ±3.5%. The 3-year cumulative incidence of chronic graft versus host disease (cGVHD) was 80. 3% ± 4. 9%. After a median follow-up of 28. 7 months, 29patients died. The 3-year estimated overall survival (OS) and disease-free survival (DFS) rates were 69. 9% ± 5.0% and 58.0% ± 5.4% respectively. The cumulative relapse rate (RR) was 25.9% ±4. 7% , while non-relapse mortality (NRM) was 16.1% ± 4.0%. Multivariate analyses showed that non Ⅱ-Ⅳ aGVHD and cGVHD were favorable factors associated with OS. Low DFS rate was correlated with high scores of international prognostic scoring system (IPSS). Patients with RAEB-t and AML (n = 31 ) were divided into 3 groups : no chemotherapy before HSCT ( Group 1 ), chemotherapy but not achieving remission ( Group 2) and chemotherapy and achieving remission (Group 3 ). The 3-year OS rate was 100. 0% in Group 3, which was significantly higher than those of Groups 1 and 2 with 33.9%, 32. 7% respectively (P 〈 0. 05). The difference of DFS and RR in the three groups did not reach statistic difference. Conclusions Allo- HSCT from HLA-identical siblings is effective for patients with MDS. IPSS is of prognostic value for post- transplantation outcome. For patients with progressive disease before transplantation, maximal control of blasts in bone marrow may improve the prognosis of advanced MDS.
出处
《中华内科杂志》
CAS
CSCD
北大核心
2014年第2期89-93,共5页
Chinese Journal of Internal Medicine
基金
国家自然科学基金重点项目(81230013)
中华医学会分子生物学临床应用研究(CAMB042010)
首都卫生发展科研专项(2011-4022-08)
关键词
骨髓增生异常综合征
造血干细胞移植
异基因
无病生存
总生存
Myelodysplastic syndromes
Hematopoietic stem cell transplantation, allogeneic
Disease-free survival
Overall survival