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.

在骨髓增生异常综合征 (MDS) 中，特别是在没有过多原始细胞的患者中，很难评估造血细胞移植 (HCT) 之前的疾病负担。我们评估了移植时的形态学发育不良是否可以作为与 MDS 患者移植后结果相关的疾病负担的衡量标准。我们确定了 2010 年至 2017 年期间在我们机构接受同种异体 HCT 的 84 名 MDS 患者，他们在 HCT 之前立即接受了骨髓评估。发育不良由两名对现有病理报告不知情的血液病理学家独立确定。红细胞核发育不良与 HCT 后结果相关，但与巨核细胞或髓细胞无关。与不存在红细胞核发育不良相比，存在与较低的 2 年无进展生存期相关（PFS；34 % vs 62 %，p = 0。0495）和 2 年总生存率（OS；34 % 对 62 %，p = 0.042）。在包括年龄、移植时的 IPSS-R、HCT 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）。移植前的发育异常评估可作为 MDS 疾病负担的估计，并确定移植前或移植后需要额外治疗的高风险患者。