Gupta Vikas, Kim Soyoung, Hu Zhen-Huan, Liu Ying, Aljurf Mahmoud, Bacher Ulrike, Beitinjaneh Amer, Cahn Jean-Yves, Cerny Jan, Copelan Edward, Gadalla Shahinaz M, Gale Robert Peter, Ganguly Siddhartha, George Biju, Gerds Aaron T, Gergis Usama, Hamilton Betty K, Hashmi Shahrukh, Hildebrandt Gerhard C, Kamble Rammurti T, Kindwall-Keller Tamila, Lazarus Hillard M, Liesveld Jane L, Litzow Mark, Maziarz Richard T, Nishihori Taiga, Olsson Richard F, Rizzieri David, Savani Bipin N, Seo Sachiko, Solh Melhem, Szer Jeff, Verdonck Leo F, Wirk Baldeep, Woolfrey Ann, Yared Jean A, Alyea Edwin P, Popat Uday R, Sobecks Ronald M, Scott Bart L, Nakamura Ryotaro, Saber Wael
Princess Margaret Cancer Centre, Toronto, ON, Canada.
Center for International Blood and Marrow Transplant Research (CIBMTR), Department of Medicine, and.
Blood Adv. 2020 Oct 13;4(19):4748-4757. doi: 10.1182/bloodadvances.2020002621.
Comparative outcomes of allogeneic hematopoietic cell transplantation (HCT) for BCR-ABL1- myeloproliferative neoplasms (MPNs) in blast phase (MPN-BP) vs de novo acute myeloid leukemia (AML), and AML with prior myelodysplastic syndromes (MDSs; post-MDS AML), are unknown. Using the Center for International Blood and Marrow Transplant Research (CIBMTR) database, we compared HCT outcomes in 177 MPN-BP patients with 4749 patients with de novo AML, and 1104 patients with post-MDS AML, using multivariate regression analysis in 2 separate comparisons. In a multivariate Cox model, no difference in overall survival (OS) or relapse was observed in patients with MPN-BP vs de novo AML with active leukemia at HCT. Patients with MPN-BP in remission had inferior OS in comparison with de novo AML in remission (hazard ratio [HR], 1.40 [95% confidence interval [CI], 1.12-1.76]) due to higher relapse rate (HR, 2.18 [95% CI, 1.69-2.80]). MPN-BP patients had inferior OS (HR, 1.19 [95% CI, 1.00-1.43]) and increased relapse (HR, 1.60 [95% CI, 1.31-1.96]) compared with post-MDS AML. Poor-risk cytogenetics were associated with increased relapse in both comparisons. Peripheral blood grafts were associated with decreased relapse in MPN-BP and post-MDS AML (HR, 0.70 [95% CI, 0.57-0.86]). Nonrelapse mortality (NRM) was similar between MPN-BP vs de novo AML, and MPN-BP vs post-MDS AML. Total-body irradiation-based myeloablative conditioning was associated with higher NRM in both comparisons. Survival of MPN-BP after HCT is inferior to de novo AML in remission and post-MDS AML due to increased relapse. Relapse-prevention strategies are required to optimize HCT outcomes in MPN-BP.
异基因造血细胞移植（HCT）治疗处于急变期的BCR-ABL1阴性骨髓增殖性肿瘤（MPN）与初发急性髓系白血病（AML）以及既往有骨髓增生异常综合征（MDS）的AML（MDS后AML）的比较结果尚不清楚。利用国际血液和骨髓移植研究中心（CIBMTR）数据库，我们在两项独立比较中采用多因素回归分析，比较了177例MPN急变期患者与4749例初发AML患者以及1104例MDS后AML患者的HCT结果。在多因素Cox模型中，对于HCT时患有活动性白血病的MPN急变期患者与初发AML患者，未观察到总生存期（OS）或复发率有差异。缓解期的MPN急变期患者与缓解期的初发AML患者相比，OS较差（风险比[HR]，1.40[95%置信区间[CI]，1.12 - 1.76]），原因是复发率较高（HR，2.18[95%CI，1.69 - 2.80]）。与MDS后AML相比，MPN急变期患者的OS较差（HR，1.19[95%CI，1.00 - 1.43]）且复发增加（HR，1.60[95%CI，1.31 - 1.96]）。在两项比较中，不良风险细胞遗传学均与复发增加相关。外周血移植物与MPN急变期和MDS后AML的复发减少相关（HR，0.70[95%CI，0.57 - 0.86]）。MPN急变期与初发AML之间以及MPN急变期与MDS后AML之间的非复发死亡率（NRM）相似。在两项比较中，基于全身照射的清髓性预处理均与较高的NRM相关。由于复发增加，MPN急变期患者HCT后的生存期低于缓解期的初发AML患者和MDS后AML患者。需要采取预防复发策略以优化MPN急变期患者的HCT结果。
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