MRD-directed risk stratification treatment may improve outcomes of t(8;21) AML in the first complete remission: results from the AML05 multicenter trial

• Published in: Blood 2013-05, Vol.121 (20), p.4056-4062
• Main Authors: Zhu, Hong-Hu, Zhang, Xiao-Hui, Qin, Ya-Zhen, Liu, Dai-Hong, Jiang, Hao, Chen, Huan, Jiang, Qian, Xu, Lan-Ping, Lu, Jin, Han, Wei, Bao, Li, Wang, Yu, Chen, Yu-Hong, Wang, Jing-Zhi, Wang, Feng-Rong, Lai, Yue-Yun, Chai, Jun-Yue, Wang, Li-Ru, Liu, Yan-Rong, Liu, Kai-Yan, Jiang, Bin, Huang, Xiao-Jun
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Antineoplastic Combined Chemotherapy Protocols - therapeutic use; Chromosomes, Human, Pair 21 - genetics; Chromosomes, Human, Pair 8 - genetics; Combined Modality Therapy; Consolidation Chemotherapy; Female; Hematopoietic Stem Cell Transplantation; Humans; Leukemia, Myeloid, Acute - diagnosis; Leukemia, Myeloid, Acute - genetics; Leukemia, Myeloid, Acute - mortality; Leukemia, Myeloid, Acute - therapy; Male; Middle Aged; Neoplasm, Residual; Prognosis; Remission Induction; Risk Assessment; Survival Analysis; Translocation, Genetic; Young Adult
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood-2012-11-468348

We aimed to improve the outcome of t(8;21) acute myeloid leukemia (AML) in the first complete remission (CR1) by applying risk-directed therapy based on minimal residual disease (MRD) determined by RUNX1/RUNX1T1 transcript levels. Risk-directed therapy included recommending allogeneic hematopoietic stem cell transplantation (allo-HSCT) for high-risk patients and chemotherapy/autologous-HSCT (auto-HSCT) for low-risk patients. Among 116 eligible patients, MRD status after the second consolidation rather than induction or first consolidation could discriminate high-risk relapse patients (P = .001). Allo-HSCT could reduce relapse and improve survival compared with chemotherapy for high-risk patients (cumulative incidence of relapse [CIR]: 22.1% vs 78.9%, P < .0001; disease-free survival [DFS]: 61.7% vs 19.6%, P = .001), whereas chemotherapy/auto-HSCT achieved a low relapse rate (5.3%) and high DFS (94.7%) for low-risk patients. Multivariate analysis revealed that MRD status and treatment choice were independent prognostic factors for relapse, DFS, and OS. We concluded that MRD status after the second consolidation may be the best timing for treatment choice. MRD-directed risk stratification treatment may improve the outcome of t(8;21) AML in CR1. This trial was registered at http://www.chictr.org as #ChiCTR-OCH-12002406. •Risk stratification treatment of t(8;21) acute myeloid leukemia may decrease relapse and improve long-term survival.•Allo-HSCT benefited high-risk patients, but impaired the survival of low-risk patients.