Multicenter prospective Phase II study of decitabine priming combined with low-dose idarubicin, cytarabine, and G-CSF in children with refractory or relapsed Acute Myeloid Leukemia
Abstract
Abstract Background: As of 2018, no standard salvage regimen had been established for pediatric relapsed or refractory acute myeloid leukemia (R/R AML).Methods: In this prospective, multicenter Phase II trial, 101 evaluable patients under 18 years of age with R/R AML received one course of decitabine priming followed by low-dose idarubicin, cytarabine, and G-CSF (DP-IAG) as remission reinduction therapy. The primary objective was to determine the rate of complete remission, including remission with incomplete hematologic recovery (CR/CRi).Results: Seventy-four patients (73.3%; 95% CI: 64.6–81.9%) achieved CR/CRi and four patients achieved PR. The overall response rate (ORR) was 77.2% (95% CI: 68.9–85.5%). Among them, 20 proceeded to allogeneic hematopoietic stem cell transplantation (allo-HSCT) after reinduction I. Among the remaining patients, 55 underwent a second reinduction course with either DP-IAG (n = 33) or standard induction chemotherapy (n = 22). After this, 31 proceeded directly to allo-HSCT. At a median follow-up of 36.4 months (IQR: 10.3–51.3 months), the estimated 3-year overall survival rate was 60.8% (95% CI: 55.9–65.7%). Infections were the most common non-hematologic adverse events, and three patients died from treatment-related toxicity after the first DP-IAG course.Conclusions: These results support DP-IAG as an effective and tolerable salvage regimen, providing a feasible bridge to HSCT and a new reference point for future trials in pediatric R/R AML.
Article Details
Authors (28)
Liyan Fan
1Children's Hospital of Soochow University, Suzhou, China
Xiaowen Zhai
3Department of Hematology and Oncology, Children’s Hospital of Fudan University, Fudan University, Shanghai, China
Ningling Wang
4Department of Pediatrics, The Second Affiliated Hospital of Anhui Medical University, Anhui Medical University, Hefei, China
Ning Liao
5Department of Pediatrics, The First Affiliated Hospital of Guangxi Medical University, Guangxi Medical University, Nanning, China
Peifang Xiao
1Department of Hematology and Oncology, Children’s Hospital of Soochow University, Soochow University, Suzhou, China
Feng Xu
Faculty of Pharmaceutical Sciences
Minghua Yang
State Key Laboratory of Natural Medicines and Jiangsu Key Laboratory of Bioactive Natural Product Research, School of Traditional Chinese Pharmacy
Xueju Xu
8Department of Pediatrics, The First Affiliated Hospital of Zhengzhou University, Zhengzhou University, Zhengzhou, China
Qi An
Jixia Luo
10Department of Hematology and Oncology, Kaifeng Children’s Hospital, Kaifeng, China
Liangchun Yang
Xiaojun Yuan
Yunyan He
Yong Zhuang
Hongsheng Wang
Jiangsu Provincial Key Laboratory of Dermatology, Hospital for Skin Diseases, Institute of Dermatology, Chinese Academy of Medical Sciences & Peking Union Medical College
Linhai Yang
4Department of Pediatrics, The Second Affiliated Hospital of Anhui Medical University, Anhui Medical University, Hefei, China
Weina Zhang
Guangdong-Hong Kong-Macao Joint Laboratory for Contaminants Exposure and Health, Guangdong Key Laboratory of Environmental Catalysis and Health Risk Control, Institute of Environmental Health and Pollution Control
Yufeng Liu
Key Laboratory of Artificial Structures and Quantum Control (Ministry of Education), School of Physics and Astronomy
Jie Li
Hailong He
Yi Wang
Jun Lu
Hua Jiang
Xiuli Ju
13Department of Pediatrics, Qilu Hospital of Shandong University, Shandong University, Qingdao, China
Qian-Fei Wang
16China National Center for Bioinformation, Beijing, China
Raul Ribeiro
1St. Jude Children's Research Hospital, Oncology, Memphis, United States
Cheng Cheng
Shaoyan Hu