Baranwal Anmol, Chhetri Rakchha, Yeung David, Clark Matthew, Shah Syed, Litzow Mark R, Hogan William J, Mangaonkar Abhishek, Alkhateeb Hassan B, Singhal Deepak, Cibich Alia, Bardy Peter, Kok Chung H, Hiwase Devendra K, Shah Mithun Vinod
Division of Hematology, Mayo Clinic, Rochester, MN, USA.
William J. von Leibig Center for Transplantation, Mayo Clinic, Rochester, MN, USA.
Bone Marrow Transplant. 2023 Jul;58(7):769-776. doi: 10.1038/s41409-023-01970-0. Epub 2023 Apr 3.
Therapy-related myeloid neoplasms (t-MN) are aggressive myeloid neoplasms. Factors predicting post-allogeneic stem cell transplant (alloSCT) survival are not well-known. We studied the prognostic utility of factors at: t-MN diagnosis, pre-alloSCT, and post-alloSCT. Primary endpoints were 3-year overall survival (OS), relapse incidence (RI), and non-relapse mortality (NRM). Post-alloSCT OS did not differ between t-MDS and t-AML (20.1 vs. 19.6 months, P = 1), though t-MDS had a significantly higher 3-year RI compared to t-AML (45.1% vs. 26.9%, P = 0.03). In t-MDS, the presence of monosomy 5 (HR 3.63, P = 0.006) or monosomy 17 (HR 11.81, P = 0.01) pre-alloSCT were associated with higher RI. Complex karyotype was the only factor adversely influencing survival at all the timepoints. The inclusion of genetic information yielded 2 risk-categories: high-risk defined by the presence of pathogenic variants (PV) in (TP53/BCOR/IDH1/GATA2/BCORL1) and standard-risk (remainder of the patients) with 3-year post-alloSCT OS of 0% and 64.6%, respectively (P = 0.001). We concluded that while alloSCT was curative in a subset of t-MN patients, outcomes remained poor, specifically in the high-risk category. t-MDS patients, especially those with persistent disease pre-alloSCT were at increased risk of relapse. Disease-related factors at t-MN diagnosis were the most prognostic of post-alloSCT survival; utility of factors available later in the course, was incremental.
治疗相关髓系肿瘤（t-MN）是侵袭性髓系肿瘤。预测异基因造血干细胞移植（alloSCT）后生存的因素尚不清楚。我们研究了t-MN诊断时、alloSCT前和alloSCT后的因素的预后价值。主要终点为3年总生存期（OS）、复发率（RI）和非复发死亡率（NRM）。t-MDS和t-AML的alloSCT后OS无差异（20.1个月对19.6个月，P = 1），尽管t-MDS的3年RI显著高于t-AML（45.1%对26.9%，P = 0.03）。在t-MDS中，alloSCT前存在5号染色体单体（HR 3.63，P = 0.006）或17号染色体单体（HR 11.81，P = 0.01）与较高的RI相关。复杂核型是在所有时间点均对生存产生不利影响的唯一因素。纳入遗传信息产生了2个风险类别：由（TP53/BCOR/IDH1/GATA2/BCORL1）中存在致病变异（PV）定义的高危组和标准风险组（其余患者），alloSCT后3年OS分别为0%和64.6%（P = 0.001）。我们得出结论，虽然alloSCT可治愈一部分t-MN患者，但结局仍然较差，特别是在高危类别中。t-MDS患者，尤其是alloSCT前疾病持续存在的患者复发风险增加。t-MN诊断时的疾病相关因素对alloSCT后生存的预后价值最大；病程后期可用因素的作用是渐进性的。
