Yu-Juan Xue1, Yi-Fei Cheng2, Ai-Dong Lu1, Yu Wang2, Ying-Xi Zuo1, Chen-Hua Yan2, Pan Suo2, Le-Ping Zhang3, Xiao-Jun Huang2. 1. Department of Pediatrics, Peking University People's Hospital, Peking University, Beijing, China. 2. Department of Hematology, Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China; Peking-Tsinghua Center for Life Sciences and Research Unit of Key Technique for Diagnosis and Treatments of Hematologic Malignancies, Chinese Academy of Medical Sciences, Beijing, China. 3. Department of Pediatrics, Peking University People's Hospital, Peking University, Beijing, China. Electronic address: zhangleping1964@126.com.
Abstract
BACKGROUND: The role of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for children with intermediate-risk acute myeloid leukemia (IR-AML) in first complete remission has been controversial. The present study compared the effect of chemotherapy with unmanipulated haplo-HSCT as treatment of patients with IR-AML in first complete remission (CR1). PATIENTS AND METHODS: We retrospectively analyzed the outcomes of 80 children with IR-AML and compared the effects of chemotherapy (n = 47) with those of haplo-HSCT (n = 33) as treatment in CR1. RESULTS: The 3-year overall survival, event-free survival (EFS), and cumulative incidence of relapse (CIR) was 85.4% ± 4.1%, 73.2% ± 5.0%, and 25.4% ± 4.5%, respectively. Compared with the chemotherapy group, the patients in the haplo-HSCT group had a lower CIR (P = .059) and better EFS (P = .108), but roughly equivalent overall survival (P = .841). Multivariate analysis revealed chemotherapy and minimal residual disease (MRD) of ≥ 10-3 after induction therapy as independent risk factors affecting CIR and EFS. EFS (P = .045) and CIR (P = .045) differed significantly between the 2 treatment groups in patients with MRD of ≥ 10-3 after induction therapy. CONCLUSION: Haplo-HSCT might be a feasible option for children with IR-AML in CR1, especially for patients with MRD of ≥ 10-3 after induction therapy.
BACKGROUND: The role of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for children with intermediate-risk acute myeloid leukemia (IR-AML) in first complete remission has been controversial. The present study compared the effect of chemotherapy with unmanipulated haplo-HSCT as treatment of patients with IR-AML in first complete remission (CR1). PATIENTS AND METHODS: We retrospectively analyzed the outcomes of 80 children with IR-AML and compared the effects of chemotherapy (n = 47) with those of haplo-HSCT (n = 33) as treatment in CR1. RESULTS: The 3-year overall survival, event-free survival (EFS), and cumulative incidence of relapse (CIR) was 85.4% ± 4.1%, 73.2% ± 5.0%, and 25.4% ± 4.5%, respectively. Compared with the chemotherapy group, the patients in the haplo-HSCT group had a lower CIR (P = .059) and better EFS (P = .108), but roughly equivalent overall survival (P = .841). Multivariate analysis revealed chemotherapy and minimal residual disease (MRD) of ≥ 10-3 after induction therapy as independent risk factors affecting CIR and EFS. EFS (P = .045) and CIR (P = .045) differed significantly between the 2 treatment groups in patients with MRD of ≥ 10-3 after induction therapy. CONCLUSION: Haplo-HSCT might be a feasible option for children with IR-AML in CR1, especially for patients with MRD of ≥ 10-3 after induction therapy.
Authors: Jae Min Lee; Eu Jeen Yang; Kyung Mi Park; Young-Ho Lee; Heewon Chueh; Jeong Ok Hah; Ji Kyoung Park; Jae Young Lim; Eun Sil Park; Sang Kyu Park; Heung Sik Kim; Ye Jee Shim; Jeong A Park; Eun Jin Choi; Kun Soo Lee; Ji Yoon Kim; Young Tak Lim Journal: Children (Basel) Date: 2021-02-05