Antoniou Evangelia, Puschnig Annika, Niktoreh Naghmeh, Hoffmeister Lina Marie, Schneider Markus, Augsburg Carolin, Katerkamp Christel, Kondryn Denise, Ziedrich Tabea, Klusmann Jan-Henning, Dworzak Michael, Creutzig Ursula, Mühlegger Nora, Melanie Lux, Waack Katharina, von Neuhoff Nils, Reinhardt Dirk, Sendker Stephanie
Department of Paediatric Haematology and Oncology, University Children's Hospital, Paediatric III, University Medicine Essen, Germany.
German Cancer Consortium (DKTK), Site Essen, National Centre for Tumour Diseases (NCT) Site Essen, University Hospital Essen, Essen, 45147, Germany.
EClinicalMedicine. 2025 May 31;84:103272. doi: 10.1016/j.eclinm.2025.103272. eCollection 2025 Jun.
In AML, the assessment of response is one of the most important prognostic markers and is crucial for assessing clinical trials. Remission criteria for AML defined by Cheson et al. has remained virtually unchanged over the last two decades. Here we revised response criteria and provide a more precise standard for evaluating treatment outcomes in paediatric AML.
A multicentre analysis of real-time data of 1094 paediatric patients diagnosed between 2004 and 2021 within the AML-BFM study group was conducted, categorising them into two alternative response groups "evidence of leukaemia" (EL, ≥5% blasts) and "no evidence of leukaemia" (NEL, <5% blasts) and comparing those with the previous CR versus no-CR classification across three different treatment timepoints.
The survival of patients newly classified as NEL was comparable to that of patients with CR. The revised NEL/EL comparison showed better predictive power in terms of survival than the former CR/no-CR classification. The poorest outcome at the end of induction (EOI) was noticed in patients with EL, which could be improved by HSCT. Stratification by NEL/EL revealed the highest independent predictive effect on overall and event-free survival. The integration of measurable residual disease monitoring using reverse transcriptase quantitative polymerase chain reaction (RT-qPCR) further improved the predictive power of the revised definition.
By simplifying the response criteria for morphological assessment of blast persistence (NEL/EL), we provide an improved and more precise definition of response for paediatric AML, which presents a new milestone with a high prognostic impact in daily clinical practice and in interpreting clinical trials.
Deutsche Krebshilfe e.V., University of Duisburg-Essen.
在急性髓系白血病（AML）中，缓解评估是最重要的预后标志物之一，对评估临床试验至关重要。Cheson等人定义的AML缓解标准在过去二十年中几乎没有变化。在此，我们修订了缓解标准，并为评估儿童AML的治疗结果提供了更精确的标准。
对AML-BFM研究组在2004年至2021年期间诊断的1094例儿科患者的实时数据进行了多中心分析，将他们分为两个替代缓解组：“白血病证据”（EL，≥5%原始细胞）和“无白血病证据”（NEL，<5%原始细胞），并在三个不同的治疗时间点将其与之前的完全缓解（CR）与未缓解分类进行比较。
新分类为NEL的患者生存率与CR患者相当。修订后的NEL/EL比较在生存预测方面比之前的CR/未缓解分类显示出更好的预测能力。诱导结束时（EOI）预后最差的是EL患者，异基因造血干细胞移植（HSCT）可改善其预后。按NEL/EL分层显示对总生存和无事件生存具有最高的独立预测作用。使用逆转录定量聚合酶链反应（RT-qPCR）进行可测量残留病监测的整合进一步提高了修订定义的预测能力。
通过简化原始细胞持续存在的形态学评估缓解标准（NEL/EL），我们为儿童AML提供了一个改进的、更精确的缓解定义，这在日常临床实践和解释临床试验中具有高预后影响，是一个新的里程碑。
德国癌症援助基金会、杜伊斯堡-埃森大学
Zotero 插件
