Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY.
Department of Medicine, University College Hospital, London, United Kingdom.
Blood Adv. 2023 Aug 8;7(15):3993-4002. doi: 10.1182/bloodadvances.2023009886.
To develop a prognostic model for patients undergoing allogeneic hematopoietic cell transplantation (allo-HCT) for myelofibrosis (MF), we examined the data of 623 patients undergoing allo-HCT between 2000 and 2016 in the United States (the Center for International Blood and Marrow Transplant Research [CIBMTR] cohort). A Cox multivariable model was used to identify factors prognostic of mortality. A weighted score using these factors was assigned to patients who received transplantation in Europe (the European Bone Marrow Transplant [EBMT] cohort; n = 623). Patient age >50 years (hazard ratio [HR], 1.39; 95% confidence interval [CI], 0.98-1.96), and HLA-matched unrelated donor (HR, 1.29; 95% CI, 0.98-1.7) were associated with an increased hazard of death and were assigned 1 point. Hemoglobin levels <100 g/L at time of transplantation (HR, 1.63; 95% CI, 1.2-2.19) and a mismatched unrelated donor (HR, 1.78; 95% CI, 1.25-2.52) were assigned 2 points. The 3-year overall survival (OS) in patients with a low (1-2 points), intermediate (3-4 points), and high score (5 points) were 69% (95% CI, 61-76), 51% (95% CI, 46-56.4), and 34% (95% CI, 21-49), respectively (P < .001). Increasing score was predictive of increased transplant-related mortality (TRM; P = .0017) but not of relapse (P = .12). The derived score was predictive of OS (P < .001) and TRM (P = .002) but not of relapse (P = .17) in the EBMT cohort as well. The proposed system was prognostic of survival in 2 large cohorts, CIBMTR and EBMT, and can easily be applied by clinicians consulting patients with MF about the transplantation outcomes.
为了为接受异基因造血细胞移植(allo-HCT)治疗骨髓纤维化(MF)的患者开发预后模型,我们检查了 2000 年至 2016 年间在美国接受 allo-HCT 的 623 名患者的数据(国际血液和骨髓移植研究中心 [CIBMTR] 队列)。使用 Cox 多变量模型确定与死亡率相关的预后因素。使用这些因素为在欧洲接受移植的患者(欧洲骨髓移植 [EBMT] 队列;n=623)分配加权评分。患者年龄>50 岁(风险比 [HR],1.39;95%置信区间 [CI],0.98-1.96)和 HLA 匹配的无关供体(HR,1.29;95%CI,0.98-1.7)与死亡风险增加相关,并被分配 1 分。移植时血红蛋白水平<100 g/L(HR,1.63;95%CI,1.2-2.19)和不匹配的无关供体(HR,1.78;95%CI,1.25-2.52)被分配 2 分。低(1-2 分)、中(3-4 分)和高(5 分)评分患者的 3 年总生存率(OS)分别为 69%(95%CI,61-76)、51%(95%CI,46-56.4)和 34%(95%CI,21-49)(P<.001)。评分增加预示着移植相关死亡率(TRM;P=.0017)增加,但不预示复发(P=.12)。该评分系统在 EBMT 队列中也能预测 OS(P<.001)和 TRM(P=.002),但不能预测复发(P=.17)。该系统在 CIBMTR 和 EBMT 两个大型队列中均具有预后生存能力,临床医生可在咨询 MF 患者移植结果时轻松应用。