Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, Maryland.
Division of Biostatistics and Bioinformatics, Johns Hopkins/Sidney Kimmel Comprehensive Cancer Center, Baltimore, Maryland.
Transplant Cell Ther. 2022 May;28(5):259.e1-259.e11. doi: 10.1016/j.jtct.2022.02.004. Epub 2022 Feb 11.
We describe outcomes after post-transplantation cyclophosphamide and nonmyeloablative conditioning-based allogeneic blood or marrow transplantation for myelofibrosis using matched or mismatched related or unrelated donors. The conditioning regimen consisted of fludarabine, cyclophosphamide, and total body irradiation. Forty-two patients were included, with a median age of 63 years, of whom 19% had Dynamic International Prognostic Scoring System (DIPSS)-plus intermediate-1 risk, 60% had intermediate-2 risk, and 21% had high-risk disease, and 60% had at least 1 high-risk somatic mutation. More than 90% of patients engrafted neutrophils, at a median of 19.5 days, and 7% experienced graft failure. At 1 year and 3 years, respectively, overall survival was 65% and 60%, relapse-free survival was 65% and 31%, relapse was 5% and 40%, and nonrelapse mortality was 30% and 30%. Acute graft-versus-host disease grade 3-4 was seen in 17% of patients at 1 year, and chronic graft-versus-host disease requiring systemic therapy in occurred in 12% patients. Spleen size ≥17 cm or prior splenectomy was associated with inferior relapse-free survival (hazard ratio [HR], 3.50; 95% confidence interval [CI], 1.18 to 10.37; P = .02) and higher relapse rate (subdistribution HR [SDHR] not calculable; P = .01). Age >60 years (SDHR, 0.26; 95% CI, 0.08 to 0.80, P = .02) and receipt of peripheral blood grafts (SDHR, 0.34; 95% CI, 0.11 to 0.99; P = .05) were associated with a lower risk of relapse. In our limited sample, the presence of a high-risk mutation was not statistically significantly associated with an inferior outcome, although ASXL1 was suggestive of inferior survival (SDHR, 2.36; 95% CI, 0.85 to 6.6; P = .09). Overall, this approach shows outcomes comparable those of to previously reported approaches and underscores the importance of spleen size in the evaluation of transplantation candidates.
我们描述了使用匹配或不匹配的相关或无关供体进行移植后环磷酰胺和非清髓性预处理的异基因血液或骨髓移植治疗骨髓纤维化的结果。预处理方案包括氟达拉滨、环磷酰胺和全身照射。共纳入 42 例患者，中位年龄为 63 岁，其中 19%为动态国际预后评分系统（DIPSS）+中间-1 风险，60%为中间-2 风险，21%为高危疾病，60%至少有 1 个高危体细胞突变。超过 90%的患者中性粒细胞植入，中位数为 19.5 天，7%发生移植物失败。分别在 1 年和 3 年时，总生存率为 65%和 60%，无复发生存率为 65%和 31%，复发率为 5%和 40%，非复发死亡率为 30%和 30%。1 年内有 17%的患者出现急性移植物抗宿主病 3-4 级，12%的患者发生需要系统治疗的慢性移植物抗宿主病。脾脏大小≥17cm 或脾切除与无复发生存率降低相关（风险比[HR]，3.50；95%置信区间[CI]，1.18 至 10.37；P=0.02）和更高的复发率（亚分布 HR [SDHR] 不可计算；P=0.01）。年龄>60 岁（SDHR，0.26；95%CI，0.08 至 0.80，P=0.02）和接受外周血移植物（SDHR，0.34；95%CI，0.11 至 0.99；P=0.05）与较低的复发风险相关。在我们的有限样本中，高危突变的存在与不良结局没有统计学显著相关，但 ASXL1 提示生存率较低（SDHR，2.36；95%CI，0.85 至 6.6；P=0.09）。总体而言，这种方法显示出与先前报道的方法相当的结果，并强调了脾脏大小在评估移植候选者中的重要性。
