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异体造血干细胞移植治疗低危骨髓增生异常综合征。

Allogeneic Stem Cell Transplantation for Patients with Lower-Risk Myelodysplastic Syndrome.

机构信息

Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany; Department of Hematology, University Medical Center Ljubljana, Ljubljana, Slovenia.

Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.

出版信息

Biol Blood Marrow Transplant. 2020 Nov;26(11):2047-2052. doi: 10.1016/j.bbmt.2020.07.018. Epub 2020 Jul 24.

DOI:10.1016/j.bbmt.2020.07.018
PMID:32717435
Abstract

The indication for allogeneic stem cell transplantation (SCT) in patients with lower-risk myelodysplastic syndrome (MDS) is controversial. Here we report 60 patients with a low risk (n = 32) or intermediate risk (n = 28) classification according to the revised International Prognostic Scoring System (IPSS-R) who underwent allogeneic SCT with a reduced-intensity conditioning (n = 45) or myeloablative conditioning (n = 15) regimen from an HLA-identical sibling (n = 9), a matched unrelated donor (n = 36), or a mismatched unrelated donor (n = 15). The rates of grade II-IV and grade III-IV acute graft-versus-host disease were 32% and 7%, respectively, resulting in a transplantation-related mortality (TRM) of 17% at 3 years. The cumulative incidence of relapse at 5 years was only 7%, resulting in a 5-year disease-free survival of 72% and overall survival (OS) of 79%. Transplantation from a fully matched donor resulted in significantly improved OS at 5 years (91% versus 70%). Allogeneic SCT in lower-risk MDS (IPSS-R low or intermediate risk) from an HLA-matched donor resulted in excellent OS with a low risk of relapse.

摘要

异基因造血干细胞移植(SCT)在低危骨髓增生异常综合征(MDS)患者中的适应证存在争议。我们在此报告了 60 例低危(n=32)或中危(n=28)根据修订后的国际预后评分系统(IPSS-R)分类的患者,他们接受了异基因 SCT,预处理方案为低强度(n=45)或清髓性(n=15),供者为 HLA 匹配的同胞(n=9)、匹配的无关供者(n=36)或不匹配的无关供者(n=15)。Ⅱ-Ⅳ级和Ⅲ-Ⅳ级急性移植物抗宿主病的发生率分别为 32%和 7%,导致 3 年时移植相关死亡率(TRM)为 17%。5 年时复发的累积发生率仅为 7%,导致 5 年无病生存率为 72%和总生存率(OS)为 79%。来自完全匹配供者的移植可显著改善 5 年 OS(91% vs. 70%)。来自 HLA 匹配供者的低危 MDS(IPSS-R 低危或中危)患者进行异基因 SCT 可获得极好的 OS 且复发风险低。

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