Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota.
Division of Hematology, Mayo Clinic, Rochester, Minnesota.
Transplant Cell Ther. 2023 Jun;29(6):360.e1-360.e8. doi: 10.1016/j.jtct.2023.02.002. Epub 2023 Feb 9.
Allogeneic stem cell transplantation (alloSCT) is the only known curative treatment for myelofibrosis (MF). Risk assessment remains important for patient counseling and predicting survival outcomes for relapse and nonrelapse mortality (NRM). Outcome-prediction tools can guide decision-making. Their use in MF has relied on their extrapolation from other malignancies. The primary objective of this study was to assess the performance of the Hematopoietic cell Transplantation Comorbidity Index (HCT-CI), the augmented HCT-CI (aHCT-CI), and the Endothelial Activation and Stress Index (EASIX) in predicting NRM in patients with MF undergoing alloSCT. We retrospectively reviewed patients with MF undergoing alloSCT between 2012 and 2020 at the Mayo Clinic. Data were abstracted from the electronic medical record. EASIX score was calculated before starting conditioning therapy and analyzed based on log2- transformed values. We evaluated the log2-EASIX scores by quartiles to assess the effect of increasing values on NRM. NRM was evaluated using competing risk analyses. We used the Kaplan-Meier and log-rank methods to evaluate OS. The Fine-Gray model was used to determine risk factors for NRM. The performance of HCT-CI and aHCT-CI was compared by evaluation of model concordance given the high correlation between HCT-CI and aHCT-CI (r = .75). A total of 87 patients were evaluated. The median duration of follow-up after alloSCT was 5 years (95% confidence interval [CI], 4.4 to 6.31 years). Patients with a high HCT-CI score had significantly increased cumulative incidence of NRM at 3 years (35.5% versus 11.6%; P = .011) after alloSCT. A progressively increasing 3-year NRM was observed with increasing aHCT-CI risk category, and patients with a high or very high aHCT-CI score had significantly higher 3-year NRM compared to those with intermediate-risk or low-risk aHCT-CI scores at 3 years post-alloSCT (31.9% versus 6.52%; P = .004). An increasing log2-EASIX score quartile was not associated with 3-year NRM (19.0% versus 10.1% versus 25% versus 14.3%; P = .59), and the EASIX score was not found to be a predictor of post-transplantation NRM. A high HCT-CI was associated with significantly worse 3-year overall survival (OS) (hazard ratio [HR], 4.41; 95% CI, 1.97 to 9.87; P < .001). A high or very high aHCT-CI was significantly associated with poor 3-year OS (HR, 3.99; 95% CI, 1.56 to 10.22; P = .004). An increasing log2-EASIX score quartile group was not associated with 3-year OS (3-year OS rate, 66.7% versus 80.4% versus 64.6% versus 76.2%; P = .57). The EASIX score should not be used routinely in patients with MF. Both the HCT-CI and the aHCT-CI are accurate in predicting long-term survival outcomes in this patient population. Further studies are important to validate our findings of the role of EASIX in predicting NRM in patients with MF or other myeloproliferative neoplasms undergoing alloSCT. © 2023 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc.
异基因造血干细胞移植(alloSCT)是骨髓纤维化(MF)唯一已知的治愈性治疗方法。风险评估对于患者咨询和预测复发和非复发死亡率(NRM)的生存结果仍然很重要。预后预测工具可以指导决策。它们在 MF 中的应用依赖于从其他恶性肿瘤推断而来。本研究的主要目的是评估造血细胞移植合并症指数(HCT-CI)、增强的 HCT-CI(aHCT-CI)和内皮激活和应激指数(EASIX)在预测接受 alloSCT 的 MF 患者 NRM 中的表现。我们回顾性分析了 2012 年至 2020 年在梅奥诊所接受 alloSCT 的 MF 患者。从电子病历中提取数据。EASIX 评分在开始预处理治疗前计算,并根据对数 2 转换值进行分析。我们通过四分位法评估 log2-EASIX 评分,以评估增加值对 NRM 的影响。使用竞争风险分析评估 NRM。我们使用 Kaplan-Meier 和对数秩方法评估 OS。使用 Fine-Gray 模型确定 NRM 的危险因素。由于 HCT-CI 和 aHCT-CI 之间存在高度相关性(r =.75),因此通过评估模型一致性来比较 HCT-CI 和 aHCT-CI 的性能。共评估了 87 例患者。alloSCT 后中位随访时间为 5 年(95%置信区间[CI],4.4 至 6.31 年)。alloSCT 后 3 年,高 HCT-CI 评分患者的 NRM 累积发生率显著增加(35.5%比 11.6%;P =.011)。随着 aHCT-CI 风险类别的增加,观察到 3 年 NRM 呈渐进性增加,与中危或低危 aHCT-CI 评分患者相比,高危或极高危 aHCT-CI 评分患者在 alloSCT 后 3 年的 NRM 显著更高(31.9%比 6.52%;P =.004)。增加的 log2-EASIX 评分四分位数与 3 年 NRM 无关(19.0%比 10.1%比 25%比 14.3%;P =.59),EASIX 评分不是移植后 NRM 的预测因素。高 HCT-CI 与 3 年总生存(OS)显著较差相关(风险比[HR],4.41;95%CI,1.97 至 9.87;P <.001)。高或极高 aHCT-CI 与 3 年 OS 不良显著相关(HR,3.99;95%CI,1.56 至 10.22;P =.004)。增加的 log2-EASIX 评分四分位组与 3 年 OS 无关(3 年 OS 率,66.7%比 80.4%比 64.6%比 76.2%;P =.57)。EASIX 评分不应常规用于 MF 患者。HCT-CI 和 aHCT-CI 都能准确预测该患者人群的长期生存结果。进一步的研究对于验证我们在接受 alloSCT 的 MF 或其他骨髓增生性肿瘤患者中 EASIX 在预测 NRM 中的作用的发现很重要。