Given the heterogeneity of acute myeloid leukemia patients, it is necessary to identify patients considered fit for intensive therapy but who will perform poorly, and in whom alternative approaches deserve investigation. We analyzed 1034 fit adults ≤70 years intensively treated between 2012 and 2022 in the CETLAM group. Young adults ( ≤ 60 years) presented higher remission rates and improved survival than older adults above that age (CR 79% vs. 73%; p = 0.03 and 4-yr OS 53% vs. 33%; p < 0.001). Remission and survival outcomes varied among different genetic subsets. An especially adverse genetic group included complex, monosomal karyotype, TP53 alterations (deleted/mutated), and MECOMr. Transplant feasibility in this very adverse risk group was low, and OS and EFS at 4 years were 14% and 12%, in contrast to 70% and 57% in the favorable group and 38% and 32% in all other patients. We integrated clinical and genetic data into the Intensive Chemotherapy Score for AML (ICSA) with 6-risk categories with significantly different remission rates and OS, validated in another cohort of 581 AML patients from a previous CETLAM protocol. In summary, we identified groups of fit patients that benefit differently from an intensive approach which may be helpful in future treatment decisions.
鉴于急性髓系白血病患者的异质性，有必要识别那些虽适合强化治疗但预后不佳的患者群体，以便探索替代治疗方案。本研究分析了CETLAM组2012年至2022年间接受强化治疗的1034例≤70岁适宜成年患者。较年轻患者（≤60岁）比较年长患者（>60岁）展现出更高的缓解率和生存率（完全缓解率79% vs. 73%；p=0.03；4年总生存率53% vs. 33%；p<0.001）。不同遗传亚组的缓解与生存结果存在显著差异：具有复杂核型、单体核型、TP53改变（缺失/突变）及MECOM重排的遗传亚组预后极差。该极高危组的移植可行性较低，其4年总生存率和无事件生存率仅为14%和12%，而低危组分别为70%和57%，其他所有患者为38%和32%。我们将临床与遗传数据整合为急性髓系白血病强化化疗评分系统，划分出6个风险等级，各等级间缓解率与总生存率差异显著，该评分系统已在早期CETLAM方案的581例AML患者队列中得到验证。总之，本研究明确了从强化治疗方案中获益程度不同的适宜患者亚群，这可能为未来治疗决策提供参考。
肿瘤(癌症)患者之家