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2905 Decitabine Combined with Low-Dose Chemotherapy Is Effective in the Treatment of Pediatric Refractory/Relapsed Acute Myeloid Leukemia

Program: Oral and Poster Abstracts
Session: 615. Acute Myeloid Leukemias: Commercially Available Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster II
Hematology Disease Topics & Pathways:
Research, clinical trials, Clinical Research
Sunday, December 10, 2023, 6:00 PM-8:00 PM

Liyan Fan1*, Hua Jiang, MD PhD2*, Hongjun Liu, MD3*, Ning Liao, MD4*, Ningling Wang, MD5*, Xiaowen Zhai, MD PhD6*, Xiuli Ju, MD PhD7*, Yufeng Liu, MD PhD8*, Jixia Luo, MD9*, Peifang Xiao1*, Jun Lu1*, Hailong He1*, Yi Wang1*, Raul C. Ribeiro, MD10 and Shaoyan Hu, MD, PhD1

1Department of Hematology and Oncology, Children's Hospital of Soochow University, Suzhou, China
2Department of Hematology and Oncology, Guangzhou Women and Children's Medical Center, Guangzhou, China
3Department of Hematology and Oncology, Anhui Provincial Hospital, University of Science and Technology of China, Hefei, China
4Department of Pediatrics, The First Affiliated Hospital of Guangxi Medical University, Nanning, China
5Department of Hematology and Oncology, The Second Affiliated Hospital of Anhui Medical University, Hefei, China
6Department of Hematology and Oncology, Children's Hospital of Fudan University, Shanghai, China
7Department of Hematology and Oncology, Qilu Hospital of Shandong University, Qingdao, China
8Department of Hematology and Oncology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, China
9Department of Hematology and Oncology, Kaifeng Children's Hospital, Kaifeng, China
10Division of Leukemia/Lymphoma, Department of Oncology, St. Jude Children's Research Hospital, Memphis, TN

Background: Refractory and relapsed acute myeloid leukemia are usually associated with poor outcomes. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only curative option for children suffering from refractory/relapsed AML. Complete remission before transplantation can reduce the recurrence rate after transplantation and improve the survival rate. Previous studies have found that low-dose chemotherapy has a complete response rate and long-term survival rate comparable to standard dose chemotherapy, and chemotherapy-related toxicities are lower. Decitabine is active in both initial induction therapy and post-relapse therapy of AML, and has a synergistic effect on apoptosis of leukemia cells when combined with cytarabine. We therefore conducted a prospective study to confirm the efficacy of decitabine combined with low-dose chemotherapy in the treatment of pediatric refractory/relapsed AML. The trial is registered under ChiCTR1800015872(A multicenter clinical study of decitabine combined with low-dose chemotherapy in the treatment of refractory/relapsed AML in children).

Methods: We randomized 89 participants < 18 years with refractory/relapsed AML to receive decitabine combined with low-dose chemotherapy. All patients received decitabine 20 mg/ (m2·d) ×5 days, intravenous; combined with idarubicin 5 mg/ (m2·d), qod×3 times, intravenous; cytarabine 10 mg/ (m2·d), q12h×10 days, subcutaneous; granulocyte stimulating factor 5μg/ (kg·d), qd×10 days, intravenous. Subsequently, children received additional intensive chemotherapy or hematopoietic stem cell transplantation (HSCT) as post-remission consolidation. The primary endpoint was overall survival.

Results: From June 2018 to June 2022,the trial included 31 children with relapsed AML and 58 children with refractory AML. The male to female ratio was 57:32, median age 100 months (range 59-133months). The most common fusion genes were AML-ETO(17, 19.1%) and MLL-r (17, 19.1%), followed by NUP98- (7, 7.9%),CBFβ-MYH11(5, 5.6%). The most common mutation was WT1(18,20.2%), while FLT3, NRAS, and PTPN11 had the same proportions. (14, 15.7%).After one course of chemotherapy, 48 children (53.9%) achieved CR/CRi, 21 children (23.6%) achieved PR, and 20 children (22.5%) achieved NR, with an overall response rate of 77.5%. Thirty-one of these children received a second course of decitabine combined with low-dose chemotherapy. 11 children (35.5%) achieved CR/CRi, 17 children (54.8%) achieved PR, and 3 children (9.7%) remained NR. Of the 89 children, 66 (74.2%) children eventually received HSCT ,while the remaining 23 did not. The five-year cumulative survival rate was 64.7%. Among them, the five-year survival rate of relapsed AML is 52.6%,while that of refractory AML was 76.1%. The survival rate of refractory AML was better, but there was no statistical difference in P value between the two groups. The five-year survival rate was 82.7% for those who received HSCT, compared with 19.8% for those who only received chemotherapy, the P-value was statistically significant. The survival rate of different fusion genes was shown in figure 1.

Conclusions: Decitabine combined with low-dose chemotherapy is a favorable benefit-risk profile and may be a promising option for refractory/relapsed AML in children. Hematopoietic stem cell transplantation after remission can improve survival rate.

Disclosures: No relevant conflicts of interest to declare.

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