Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Department of Hematopathology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Cancer. 2023 Apr 1;129(7):1017-1029. doi: 10.1002/cncr.34609. Epub 2023 Jan 30.
The aim of this study was to develop a prognostic model for survival in older/unfit patients with newly diagnosed acute myeloid leukemia (AML) who were treated with lower-intensity chemotherapy regimens.
The authors reviewed all older/unfit patients with newly diagnosed AML who received lower-intensity chemotherapy from 2000 until 2020 at their institution. A total of 1462 patients were included. They were divided (3:1 basis) into a training (n = 1088) and a validation group (n = 374).
In the training cohort of 1088 patients (median age, 72 years), the multivariate analysis identified 11 consistent independent adverse factors associated with survival: older age, therapy-related myeloid neoplasm, existence of previous myelodysplastic syndrome or myeloproliferative neoplasms, poor performance status, pulmonary comorbidity, anemia, thrombocytopenia, elevated lactate dehydrogenase, cytogenetic abnormalities, and the presence of infection at diagnosis, and therapy not containing venetoclax. The 3-year survival rates were 52%, 24%, 10%, and 1% in favorable, intermediate, poor, and very poor risk, respectively. This survival model was validated in an independent cohort. In a subset of patients in whom molecular mutation profiles were performed in more recent times, adding the mutation profiles after accounting for the effects of previous factors identified IDH2 (favorable), NPM1 (favorable), and TP53 (unfavorable) mutations as molecular prognostic factors.
The proposed survival model with lower-intensity chemotherapy in older/unfit patients with newly diagnosed AML may help to advise patients on their expected outcome, to propose different strategies in first complete remission, and to compare the results of different existing or future investigational therapies.
Lower intensity therapy can be considered for older patients to avoid severe toxicities and adverse events. However, survival prediction in AML was commonly developed in patients who received intensive chemotherapy. In this study, we have proposed a survival model to guide therapeutic approach in older patients who received lower-intensity therapy.
本研究旨在为接受低强度化疗方案治疗的新诊断急性髓系白血病（AML）的老年/体弱患者建立一个生存预后模型。
作者回顾了 2000 年至 2020 年期间在其机构接受低强度化疗的所有老年/体弱的新诊断 AML 患者。共纳入 1462 例患者。他们按照（3:1 的比例）分为训练组（n=1088）和验证组（n=374）。
在 1088 例患者的训练队列中（中位年龄 72 岁），多变量分析确定了 11 个与生存相关的一致独立不良因素：年龄较大、治疗相关髓系肿瘤、既往骨髓增生异常综合征或骨髓增生性肿瘤的存在、体能状态差、肺部合并症、贫血、血小板减少、乳酸脱氢酶升高、细胞遗传学异常、诊断时存在感染以及未接受 venetoclax 治疗。在有利、中等、差和非常差风险组中，3 年生存率分别为 52%、24%、10%和 1%。该生存模型在独立队列中得到验证。在最近进行了分子突变谱分析的患者亚组中，在考虑到先前因素的影响后加入突变谱，确定 IDH2（有利）、NPM1（有利）和 TP53（不利）突变为分子预后因素。
在新诊断为 AML 的老年/体弱患者中，采用低强度化疗的拟议生存模型可以帮助患者了解预期的结果，提出在首次完全缓解时的不同策略，并比较不同现有或未来的研究性治疗的结果。
对于老年患者，可以考虑采用低强度治疗以避免严重的毒性和不良事件。然而，AML 的生存预测通常是在接受强化化疗的患者中开发的。在这项研究中，我们提出了一个生存模型，以指导接受低强度化疗的老年患者的治疗方法。
