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红细胞核发育异常与接受异基因造血细胞移植的骨髓增生异常综合征患者的不良预后相关。

Erythroid nuclear dysplasia is associated with inferior outcomes for patients with myelodysplastic syndrome undergoing allogeneic hematopoietic cell transplantation.

机构信息

Department of Medicine, Massachusetts General Hospital, Boston, MA, USA.

Department of Biostatistics, Dana-Farber Cancer Institute, Boston, MA, USA.

出版信息

Leuk Res. 2021 Oct;109:106625. doi: 10.1016/j.leukres.2021.106625. Epub 2021 May 18.

Abstract

Disease burden prior to hematopoietic cell transplantation (HCT) is difficult to assess in myelodysplastic syndrome (MDS), particularly in patients without excess blasts. We assessed whether morphologic dysplasia at the time of transplant can be a metric of disease burden that is associated with post-transplant outcomes in MDS patients. We identified 84 MDS patients undergoing allogeneic HCT at our institution between 2010 and 2017 who received a bone marrow evaluation immediately prior to HCT. Dysplasia was independently determined by two hematopathologists blinded to existing pathology reports. Erythroid nuclear dysplasia, but not megakaryocytic or myeloid, was associated with post-HCT outcomes. Presence compared to absence of erythroid nuclear dysplasia was associated with lower 2-year progression-free survival (PFS; 34 % vs 62 %, p = 0.0495) and 2-year overall survival (OS; 34 % vs 62 %, p = 0.042). In a multivariate analysis including age, IPSS-R at the time of transplant, pre-HCT therapy, and donor type as covariates, erythroid nuclear dysplasia remained associated with lower PFS (HR 2.6, p = 0.036) and OS (HR 2.7, p = 0.028). Dysplasia assessment prior to transplant may serve as an estimate of disease burden in MDS and identify high-risk patients who merit additional therapies pre- or post-transplant.

摘要

在骨髓增生异常综合征(MDS)中,造血细胞移植(HCT)前的疾病负担难以评估,尤其是在没有过多原始细胞的患者中。我们评估了移植时的形态学发育不良是否可以作为疾病负担的指标,与 MDS 患者移植后的结果相关。我们确定了 2010 年至 2017 年间在我们机构接受异基因 HCT 的 84 例 MDS 患者,他们在 HCT 前立即接受了骨髓评估。两名血液病理学家独立地对发育不良进行了评估,他们对现有的病理报告并不知情。红细胞核发育不良,但不是巨核细胞或髓系发育不良,与移植后结果相关。与不存在红细胞核发育不良相比,存在红细胞核发育不良与较低的 2 年无进展生存率(PFS;34%对 62%,p=0.0495)和 2 年总生存率(OS;34%对 62%,p=0.042)相关。在包括年龄、移植时 IPSS-R、移植前治疗和供体类型作为协变量的多变量分析中,红细胞核发育不良仍然与较低的 PFS(HR 2.6,p=0.036)和 OS(HR 2.7,p=0.028)相关。移植前的发育不良评估可以作为 MDS 疾病负担的估计,并识别需要在移植前或移植后进行额外治疗的高危患者。

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