Clinical features and long-term outcomes of pediatric patients with de novo acute myeloid leukemia in China with or without specific gene abnormalities: a cohort study of patients treated with BCH-AML 2005
Hongbo He,
Jun Li,
Weijing Li,
Xiaoxi Zhao,
Tianlin Xue,
Shuguang Liu,
Ruidong Zhang,
Huyong Zheng,
Chao Gao
Affiliations
Hongbo He
Hematology Center, National Key Discipline of Pediatric Hematology, National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, People’s Republic of China
Jun Li
Department of Clinical Laboratory Center, Key Laboratory of Major Diseases in Children Ministry of Education, Beijing Children’s Hospital Capital Medical University, National Center for Children’s Health, Beijing, People’s Republic of China
Weijing Li
Hematologic Disease Laboratory, National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education; Beijing Pediatric Research Institute, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, People’s Republic of China
Xiaoxi Zhao
Department of Clinical Laboratory Center, Key Laboratory of Major Diseases in Children Ministry of Education, Beijing Children’s Hospital Capital Medical University, National Center for Children’s Health, Beijing, People’s Republic of China
Tianlin Xue
Department of Clinical Laboratory Center, Key Laboratory of Major Diseases in Children Ministry of Education, Beijing Children’s Hospital Capital Medical University, National Center for Children’s Health, Beijing, People’s Republic of China
Shuguang Liu
Department of Clinical Laboratory Center, Key Laboratory of Major Diseases in Children Ministry of Education, Beijing Children’s Hospital Capital Medical University, National Center for Children’s Health, Beijing, People’s Republic of China
Ruidong Zhang
Hematology Center, National Key Discipline of Pediatric Hematology, National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, People’s Republic of China
Huyong Zheng
Hematology Center, National Key Discipline of Pediatric Hematology, National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, People’s Republic of China
Chao Gao
Department of Clinical Laboratory Center, Key Laboratory of Major Diseases in Children Ministry of Education, Beijing Children’s Hospital Capital Medical University, National Center for Children’s Health, Beijing, People’s Republic of China
Acute myeloid leukemia (AML), which has distinct genetic abnormalities, has unique clinical and biological features. In this study, the incidence, clinical characteristics, induction treatment response, and outcomes of a large cohort of Chinese AML pediatric patients treated according to the BCH-AML 2005 protocol were analyzed. RUNX1-RUNX1T1 was the most common fusion transcript, followed by the CBFβ-MHY11 and KMT2A rearrangements. FLT3-ITD and KIT mutations are associated with unfavorable clinical features and induction responses, along with KMT2A rearrangements, DEK-NUP214, and CBF-AML. The 5-year event-free survival (EFS) and overall survival (OS) rates of our cohort were 53.9 ± 3.7% and 58.5 ± 3.6%, with the best survival found among patients with CBFβ-MYH11 and the worst survival among those with DEK-NUP214. In addition, we found that patients with FLT3-ITD mutation had adverse outcomes and that KIT mutation had a negative impact on OS in RUNX1-RUNX1T1+ patients. Furthermore, the risk classification and response to treatment after each induction block also influenced the prognosis, and HSCT after first remission could improve OS in high-risk patients. Not achieving complete remission after induction 2 was found to be an independent prognostic factor for OS and EFS. These findings indicate that genetic abnormalities could be considered stratification factors, predict patient outcomes, and imply the application of targeted therapy.
