CAJ | 학술논문

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.
목적 탐토동포전상합이기인조혈간세포이식(allo-HSCT)치료골수증생이상종합정(MDS)적료효여시궤.방법 회고분석2003년1월-2012년12월채용동포전상합allo-HSCT치료MDS급MDS전급성수성백혈병(AML) 95례.채용개량마리란+배린선알혹불체랍빈적예처리방안,행골수화/혹외주혈간세포이식.결과 95례환자중93례백세포식활,Ⅱ～Ⅳ도급성이식물항숙주병(aGVHD)루계발생솔위12.9％±3.5％;만성이식물항숙주병(cGVHD)3년루계발생솔위80.3％±4.9％.3년루계복발솔(RR)위25.9％±4.7％,비복발사망솔(NRM)위16.1％±4.0％.3년예기총생존(0S)솔급무병생존(DFS)솔분별위69.9％±5.0％화58.0％±5.4％.다인소분석현시,발생Ⅱ～Ⅳ도aGVHD화불발생cGVHD시OS적독립위험인소;국제예후적분계통(IPSS)분조시DFS적독립예후인소.장난치성빈혈반원시세포증다전화형(RAEB-t)급MDS전AML환자(31례)분위이식전미화료、화료미완해、화료완해3조,3년OS솔분별위33.9％、32.7％、100.0％,화료완해조OS솔명현고우령외량조(P＜0.05),DFS솔、RR솔차이무통계학의의.결론 동포전상합allo-HSCT시치료MDS적유효수단,IPSS가예측이식후료효,대우이식전질병진전적환자,쟁취완해후행allo-HSCT가능제고료효,단상수진일보림상대조연구.
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-Ⅳacute 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-ⅣaGVHD 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 AlloHSCT from HLA-identical siblings is effective for patients with MDS.IPSS is of prognostic value for posttransplantation outcome.For patients with progressive disease before transplantation,maximal control of blasts in bone marrow may improve the prognosis of advanced MDS.