Next-Generation Sequencing is needed for the accurate genetic risk stratification of acute myeloid leukemia according to European LeukemiaNet (ELN) guidelines. We validated and compared the 2022 ELN risk classification in a real-life cohort of 546 intensively and 379 non-intensively treated patients. Among fit patients, those aged ≥65 years old showed worse OS than younger regardless risk classification. Compared with the 2017 classification, 14.5% of fit patients changed the risk with the 2022 classification, increasing the high-risk group from 44.3% to 51.8%. 3.7% and 0.9% FLT3-ITD mutated patients were removed from the favorable and adverse 2017 categories respectively to 2022 intermediate risk group. We suggest that midostaurin therapy could be a predictor for 3 years OS (85.2% with vs. 54.8% without midostaurin, P = 0.04). Forty-seven (8.6%) patients from the 2017 intermediate group were assigned to the 2022 adverse-risk group as they harbored myelodysplasia (MDS)-related mutations. Patients with one MDS-related mutation did not reach median OS, while patients with ≥2 mutations had 13.6 months median OS (P = 0.002). Patients with TP53 ± complex karyotype or inv(3) had a dismal prognosis (7.1 months median OS). We validate the prognostic utility of the 2022 ELN classification in a real-life setting providing supportive evidences to improve risk stratification guidelines.
根据欧洲白血病网络（ELN）指南，急性髓系白血病的精确遗传学风险分层需要采用新一代测序技术。我们在真实世界的546例强化治疗患者和379例非强化治疗患者队列中，对2022版ELN风险分层系统进行了验证与比较。在适宜强化治疗的患者中，无论风险分层如何，年龄≥65岁患者的总生存期均差于年轻患者。与2017版分类相比，14.5%的适宜治疗患者在2022版标准中风险类别发生改变，高危组比例从44.3%增至51.8%。其中3.7%和0.9%的FLT3-ITD突变患者分别从2017版的良好组和不良组调整至2022版中危组。我们认为米哚妥林治疗可作为3年总生存期的预测因素（用药组85.2% vs 未用药组54.8%，P=0.04）。47例（8.6%）2017版中危组患者因携带骨髓增生异常综合征相关突变，在2022版中被分配至高危组。携带单个MDS相关突变患者的中位总生存期未达到，而≥2个突变患者的中位总生存期为13.6个月（P=0.002）。TP53突变±复杂核型或inv(3)染色体易位患者预后极差（中位总生存期7.1个月）。本研究在真实临床场景中验证了2022版ELN风险分层系统的预后价值，为完善风险分层指南提供了支持性证据。
肿瘤(癌症)患者之家