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 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.

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Journal
Blood
Published
2025-11-03
DOI
https://doi.org/10.1182/blood-2025-5233
Primary Topic
Acute Myeloid Leukemia Research
Type
article
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article

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

Xueju Xu, Yunyan He, Jixia Luo, Xiaowen Zhai et al.
Blood
Acute Myeloid Leukemia Research
article

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

Xueju Xu, Yunyan He, Jixia Luo, Xiaowen Zhai, Hua Jiang, Shaoyan Hu, Xiuli Ju, Linhai Yang, Raul Correa Ribeiro, Cheng Cheng, Yufeng Liu, Dan Wang, Yi Wang, Ning Liao, Weina Zhang, Jie Li, Hongsheng Wang, Qi An, Liyan Fan, Jun Lu, Hailong He, Feng Xu, Yong Zhuang, Xiaojun Yuan, Qian-Fei Wang, Peifang Xiao, Liangchun Yang, Ningling Wang
article en

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.

BloodVol. 146(Supplement 1)
University of Science and Technology of China (CN), St. Jude Children's Research Hospital (US), Central South University (CN), Guangxi Medical University (CN), Anhui Medical University (CN), XinHua Hospital (CN), Soochow University (CN), Guangzhou Women and Children Medical Center (CN), First Affiliated Hospital of GuangXi Medical University (CN), Kaifeng City Children's Hospital (CN), Xuzhou Children Hospital (CN), Obstetrics and Gynecology Hospital of Fudan University (CN), Second Affiliated Hospital of Anhui Medical University (CN), Children's Hospital of Suzhou University (CN), First Affiliated Hospital of Zhengzhou University (CN), Third Xiangya Hospital (CN), Children's Hospital of Fudan University (CN), Xiangya Hospital Central South University (CN), Qilu Hospital of Shandong University (CN), Shanghai Center For Bioinformation Technology (CN), Hefei First People's Hospital (CN), Guangzhou Medical University (CN)
Openalex Percentile: Top 43%
Acute Myeloid Leukemia Research
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