Li-Peng Liu1, Xiao-Juan Chen1, Wen-Yu Yang1, Mei-Hui Yi1, Kang Zhou2, Min Ruan1, Fang Liu1, Xia Chen1, Li-Xian Chang1, Tian-Feng Liu1, Li Zhang1, Yao Zou1, Yu-Mei Chen1, Feng-Kui Zhang2, Xiao-Fan Zhu3, Ye Guo1. 1. Division of Pediatric Blood Diseases Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, 300020, Tianjin, China. 2. Division of of Anemia Therapeutic Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, 300020, Tianjin, China. 3. Division of Pediatric Blood Diseases Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, 300020, Tianjin, China. xfzhu@ihcams.ac.cn.
Abstract
BACKGROUND: In severe aplastic anemia (SAA), predictive markers of response to immunosuppressive therapy (IST) of porcine antilymphocyte globulin (pALG) have not been well defined. We investigated whether clinical and laboratory findings before treatment could predict response in a pediatric cohort. METHODS: In this study, we included 70 newly diagnosed SAA children and treated them with pALG. The response rate was documented during follow-up. The log-rank test compared response rates between the potential predictive factors. RESULTS: The response rate was 57.1% at 24 months follow-up. In log-rank test, mild disease severity was the most significant predictive marker of better response (P < 0.001); SAA patients with higher absolute reticulocyte count (ARC) and platelet level showed a higher response rate (both P < 0.001). Although insignificantly, elderly children and male sex show better response rate after treatment. The response rate worsened when the time interval before IST was more than 60 days. CONCLUSION: Modified IST with pALG was suitable for SAA children, and favorable response correlates with mild disease severity was identified. ARC and platelet status also appeared to be a reproducible prognostic model for response rate. IST should be started as soon as possible, given that the response rate worsens as the interval between diagnosis and treatment increases.
BACKGROUND: In severe aplastic anemia (SAA), predictive markers of response to immunosuppressive therapy (IST) of porcine antilymphocyte globulin (pALG) have not been well defined. We investigated whether clinical and laboratory findings before treatment could predict response in a pediatric cohort. METHODS: In this study, we included 70 newly diagnosed SAAchildren and treated them with pALG. The response rate was documented during follow-up. The log-rank test compared response rates between the potential predictive factors. RESULTS: The response rate was 57.1% at 24 months follow-up. In log-rank test, mild disease severity was the most significant predictive marker of better response (P < 0.001); SAApatients with higher absolute reticulocyte count (ARC) and platelet level showed a higher response rate (both P < 0.001). Although insignificantly, elderly children and male sex show better response rate after treatment. The response rate worsened when the time interval before IST was more than 60 days. CONCLUSION: Modified IST with pALG was suitable for SAAchildren, and favorable response correlates with mild disease severity was identified. ARC and platelet status also appeared to be a reproducible prognostic model for response rate. IST should be started as soon as possible, given that the response rate worsens as the interval between diagnosis and treatment increases.
Authors: X R Hu; X Zhao; L Zhang; L P Jing; W R Yang; Y Li; L Ye; K Zhou; J P Li; G X Peng; H H Fan; Y Li; Y Yang; Y Z Xiong; F K Zhang Journal: Zhonghua Xue Ye Xue Za Zhi Date: 2022-05-14