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异基因造血干细胞移植治疗骨髓增生异常综合征。

Unrelated cord blood transplantation after myeloablative conditioning in adults with advanced myelodysplastic syndromes.

机构信息

Department of Hematology and Oncology, Institute of Medical Science, University of Tokyo, Minato-ku, Tokyo, Japan.

出版信息

Bone Marrow Transplant. 2011 Feb;46(2):257-61. doi: 10.1038/bmt.2010.91. Epub 2010 Apr 19.

Abstract

We analyzed the disease-specific outcomes of adult patients with advanced myelodysplastic syndrome (MDS) treated with cord blood transplantation (CBT) after myeloablative conditioning. Between August 1998 and June 2009, 33 adult patients with advanced MDS were treated with unrelated CBT. The diagnoses at transplantation included refractory anemia with excess blasts (n=7) and MDS-related secondary AML (sAML) (n=26). All patients received four fractionated 12 Gy TBI and chemotherapy as myeloablative conditioning. The median age was 42 years, the median weight was 55 kg and the median number of cryopreserved nucleated cells was 2.51 × 10(7) cells per kg. The cumulative incidence of neutrophil recovery at day 50 was 91%. Neutrophil recovery was significantly faster in sAML patients (P=0.04). The cumulative incidence of plt recovery at day 200 was 88%. Plt recovery was significantly faster in CMV seronegative patients (P<0.001). The cumulative incidence of grade II-IV acute GVHD (aGVHD) and extensive-type chronic GVHD was 67 and 34%, respectively. Degree of HLA mismatch had a significant impact on the incidence of grade II-IV aGVHD (P=0.021). TRM and relapse at 5-years was 14 and 16%, respectively. The probability of EFS at 5 years was 70%. No factor was associated with TRM, relapse and EFS. These results suggest that adult advanced MDS patients without suitable related or unrelated BM donors should be considered as candidates for CBT.

摘要

我们分析了接受清髓性预处理的异基因造血干细胞移植(CBT)治疗的成人中高危骨髓增生异常综合征(MDS)患者的疾病特异性结局。1998 年 8 月至 2009 年 6 月,我们对 33 例成人中高危 MDS 患者进行了无关供体 CBT 治疗。移植时的诊断包括难治性贫血伴原始细胞过多(n=7)和 MDS 相关继发 AML(sAML)(n=26)。所有患者均接受了四次分割 12GyTBI 和化疗作为清髓性预处理。中位年龄为 42 岁,中位体重为 55kg,中位冻存有核细胞数为 2.51×10(7)个细胞/kg。中性粒细胞恢复至第 50 天的累积发生率为 91%。sAML 患者的中性粒细胞恢复明显更快(P=0.04)。血小板恢复至第 200 天的累积发生率为 88%。CMV 阴性患者的血小板恢复明显更快(P<0.001)。Ⅱ-Ⅳ度急性移植物抗宿主病(aGVHD)和广泛型慢性移植物抗宿主病的累积发生率分别为 67%和 34%。HLA 错配程度对Ⅱ-Ⅳ度 aGVHD 的发生率有显著影响(P=0.021)。5 年时的 TRM 和复发率分别为 14%和 16%。5 年的 EFS 概率为 70%。没有任何因素与 TRM、复发和 EFS 相关。这些结果表明,没有合适的相关或无关 BM 供者的成人中高危 MDS 患者应被视为 CBT 的候选者。

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