Delineation of features, responses, outcomes, and prognostic factors in pediatric PDGFRB-positive acute lymphoblastic leukemia patients: A retrospective multicenter study
机构：[1]Department of Pediatrics, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & BloodDiseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China[2]Department of Pediatrics, Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China华中科技大学同济医学院附属同济医院[3]Department of Hematology/Oncology, KunMing Children’s Hospital, Kunming, China[4]Department of Pediatrics, Affiliated Hospital of Qingdao University, Qingdao, China[5]Department of Hematology/Oncology, Guangzhou Women and Children’s Medical Center, Guangzhou, China[6]Department of Pediatrics, Anhui Medical University Second Affiliated Hospital, Anhui, China[7]Department of Pediatrics, Union Hospital of Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China华中科技大学同济医学院附属协和医院[8]Department of Hematology/Oncology, Shanghai Children’s Medical Center, School of Medicine, Shanghai Jiao Tong University, Key Laboratory of PediatricHematology & Oncology of China Ministry of Health, Shanghai, China[9]Department of Hematology/Oncology, Children’s Hospital of Soochow University, Suzhou, China[10]Department of Hematology/Oncology, Children’s Hospital of Nanjing Medical University, Nanjing, China[11]Department of Hematology/Oncology, Jiangxi Provincial Children’s Hospital, Nanchang, China[12]Department of Pediatrics, Qilu Hospital of Shandong University, Jinan, China[13]Department of Pediatrics, Nanfang Hospital, Southern Medical University, Guangzhou, China[14]Department of Hematology/Oncology, Children’s Hospital of Fudan University, Shanghai, China[15]Department of Hematology/Oncology, Children’s Hospital Affiliated to Shanghai Jiao Tong University, Shanghai, China[16]Department of Pediatrics, Xiangya Hospital Central South University, Changsha, China[17]Department of Pediatrics, Hong Kong Children’s Hospital, The Chinese University of Hong Kong, Hong Kong Special Administrative Region, China[18]Department of Hematology/Oncology, Xi’an Northwest Women’s and Children’s Hospital, Xi’an, China[19]Department of Pharmaceutical Sciences, St. Jude Children’s Research Hospital, Memphis, Tennessee, USA[20]Departments of Oncology, Pathology, and Global Pediatric Medicine, St. Jude Children’s Research Hospital, Memphis, Tennessee, USA[21]Department of Hematology/Oncology, Children’s Hospital of Chongqing Medical University, National Clinical Research Center for Child Health and Disorders,Chongqing, China[22]Department of Pediatrics, Key Laboratory of Birth Defects and Related Disease of Women and Children, Ministry of Education, West China Second UniversityHospital, Sichuan University, Chengdu, China
Background: PDGFRB fusions in acute lymphoblastic leukemia (ALL) is rare. The authors identified 28 pediatric PDGFRB-positive ALL. They analyzed the features, outcomes, and prognostic factors of this disease. Methods: This multicenter, retrospective study included 6457 pediatric patients with newly diagnosed PDGFRB fusion ALL according to the CCCG-ALL-2015 and CCCG-ALL-2020 protocols from April 2015 to April 2022 in 20 hospitals in China. Of these patients, 3451 were screened for PDGFRB fusions. Results: Pediatric PDGFRB-positive ALL accounted for only 0.8% of the 3451 cases tested for PDGFRB. These patients included 21 males and seven females and 24 B-ALL and 4 T-ALL; the median age was 10 years; and the median leukocyte count was 29.8 x 10(9)/L at baseline. Only one patient had eosinophilia. Three patients had an IKZF1 deletion, three had chromosome 5q31-33 abnormalities, and one suffered from a complex karyotype. The 3-year event-free survival (EFS), overall survival (OS), and cumulative incidence of relapse (CIR) were 33.1%, 65.5%, and 32.1%, respectively, with a median follow-up of 25.5 months. Twenty patients were treated with chemotherapy plus tyrosine-kinase inhibitors (TKIs) and eight were treated without TKI. Complete remission (CR) rates of them were 90.0% and 63.6%, respectively, but no differences in EFS, OS, or CIR. Univariate analyses showed patients with IKZF1 deletion or measurable residual disease (MRD) >= 0.01% after induction had inferior outcomes (p < .05). Conclusions: Pediatric PDGFRB-positive ALL has a poor outcome associated with high-risk features. Chemotherapy plus TKIs can improve the CR rate, providing an opportunity for lower MRD levels and transplantation. MRD >= 0.01% was a powerful adverse prognostic factor, and stratified treatment based on MRD may improve survival for these patients.
基金：
Chinese Academy of Medical Sciences Innovation Fund for Medical Sciences [2022-I2M-1-022, 2022-I2M-CT-B-088]; Tianjin Municipal Health Commission Science and Technology Project [TJWJ2024MS001]
第一作者机构：[1]Department of Pediatrics, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & BloodDiseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
共同第一作者：
通讯作者：
推荐引用方式(GB/T 7714)：
Zhang Xiaoyan,Wang Yaqin,Tian Xin,et al.Delineation of features, responses, outcomes, and prognostic factors in pediatric PDGFRB-positive acute lymphoblastic leukemia patients: A retrospective multicenter study[J].CANCER.2024,doi:10.1002/cncr.35481.
APA：
Zhang, Xiaoyan,Wang, Yaqin,Tian, Xin,Sun, Lirong,Jiang, Hua...&Zhu, Xiaofan.(2024).Delineation of features, responses, outcomes, and prognostic factors in pediatric PDGFRB-positive acute lymphoblastic leukemia patients: A retrospective multicenter study.CANCER,,
MLA：
Zhang, Xiaoyan,et al."Delineation of features, responses, outcomes, and prognostic factors in pediatric PDGFRB-positive acute lymphoblastic leukemia patients: A retrospective multicenter study".CANCER .(2024)
医学