Whilst allogeneic hematopoietic stem cell transplantation (allo HCT) currently offers the only curative option for patients with myelodysplastic syndrome (MDS), there is still a high risk of relapse or transplant related complications. We collected data on all patients who had undergone allo HCT at our center (Copenhagen University Hospital) between 2000 and 2018. Two hundred and fifteen patients with MDS (n=196) or CMML (n=19) were included. Estimated one-year overall survival (OS) was 70.3% (95% CI: 64.2-77.0%) and the median survival was 7.7 years (95% CI: 4.7-indeterminable). There was a significant improvement in OS over time (P=.011, comparing 2000-2010, 2010-2014 and 2014-2018). Treatment was standardized throughout the study period allowing comparison between patients receiving non-myeloablative (NMA, n=124), standard myeloablative (SMA, n=36), and fludarabine and treosulfan conditioning (FluTreo, n=55). FluTreo has myeloablative properties but lower toxicity and replaced standard MA at our center in 2014. The FluTreo group was significantly older and had more comorbidity than the SMA group, but similar disease severity. One-year OS was 84.0% (95% CI, 74.3-94.9%), 58.3% (95% CI, 44.3-76.9%), and 68.3% (95% CI, 60.2-77.5%) for FluTreo, SMA, and NMA, respectively (P=.04). In univariate analysis IPSS-R (high vs low), donor sex-mismatch, and CMV status mismatch were significant factors for OS. In multivariate analysis of OS including age, IPSS-R and HCT-CI, NMA was borderline inferior to FluTreo (P=.073) whilst SMA was significantly inferior to FluTreo with a hazard ratio of 6.89 (95% CI, 2.53-18.77, P<.001). The introduction of FluTreo allowed us to administer a myeloablative regimen to a broader patient group and shows promising results.

中文翻译：

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氟达拉滨/硫丹同种异体造血干细胞移植后骨髓增生异常综合征患者的改善结果。

尽管同种异体造血干细胞移植（allo HCT）目前是骨髓增生异常综合症（MDS）患者的唯一治疗选择，但复发或移植相关并发症的风险仍然很高。我们收集了2000年至2018年间在我们中心（哥本哈根大学医院）接受过同种HCT的所有患者的数据。其中包括115例MDS（n = 196）或CMML（n = 19）。估计的一年总生存（OS）为70.3％（95％CI：64.2-77.0％），中位生存期为7.7年（95％CI：4.7不确定）。随着时间的推移，OS有了显着改善（与2000-2010年，2010-2014年和2014-2018年相比，P = .011）。在整个研究期间，治疗都是标准化的，因此可以比较接受非清髓性（NMA，n = 124），标准清髓性（SMA，n = 36），氟达拉滨和海藻糖的调理（FluTreo，n = 55）。FluTreo具有清髓性，但毒性较低，于2014年在我们中心取代了标准MA。FluTreo组比SMA组年龄更大，合并症更多，但疾病严重程度相似。FluTreo，SMA和SMA的一年OS为84.0％（95％CI，74.3-94.9％），58.3％（95％CI，44.3-76.9％）和68.3％（95％CI，60.2-77.5％） NMA，分别为（P = .04）。在单变量分析中，IPSS-R（高vs低），供体性别不匹配和CMV状态不匹配是OS的重要因素。在包括年龄，IPSS-R和HCT-CI在内的OS的多变量分析中，NMA劣于FluTreo（P = .073），而SMA明显劣于FluTreo，危险比为6.89（95％CI，2.53-18.77， P <.001）。