Efficacy and safety of eltrombopag in the treatment of severe chronic immune thrombocytopenia in children of China: A single-center observational study
机构:[1]Department of Pharmacy, Beijing Children’s Hospital, Capital Medical University, Beijing, China医技科室药学部首都医科大学附属北京儿童医院[2]Department of Pharmacy, Beijing Chuiyangliu Hospital, Beijing, China[3]Hematology Oncology Center, Beijing Children’s Hospital, Capital Medical University, Beijing, China医技科室职能科室临床流行病与循证医学中心血液中心首都医科大学附属北京儿童医院[4]Clinical Research Center, Beijing Children’s Hospital, Capital Medical University, Beijing, China医技科室职能科室临床流行病与循证医学中心临床研究中心首都医科大学附属北京儿童医院
The treatment of severe chronic immune thrombocytopenia (SCITP) in pediatric patients is challenging. We evaluated the clinical efficacy and safety of eltrombopag in children with SCITP in China. This observational study was carried out at the Hematology Oncology Center, Beijing Children's Hospital between April 2017 and July 2018. Patients with SCITP who had at least 12 weeks of eltrombopag treatment and follow-up data were included. Baseline data, such as age, drug dosage, pre-study platelet count, concomitant medications, and bleeding severity, were collected. Treatment response rates, durable response rates, bleeding events, and adverse events were assessed during eltrombopag therapy for at least 12 weeks. The median duration of eltrombopag therapy was 16 (12-48) weeks. The overall, complete, and partial response rates were 75% (15/20), 35% (7/20), and 40% (8/20), respectively. The durable response rate was 70% (14/20). No serious bleeding events or serious adverse events occurred during the study period. Eltrombopag appears to be effective and safe in children with SCITP, although additional research is needed to confirm this.
基金:
Construction of Clinical Evaluation Technology Platform for Children Demonstration New Drugs [2017ZX09304029]; Beijing Natural Science Foundation of ChinaBeijing Natural Science Foundation [7192046]
第一作者机构:[1]Department of Pharmacy, Beijing Children’s Hospital, Capital Medical University, Beijing, China
通讯作者:
通讯机构:[1]Department of Pharmacy, Beijing Children’s Hospital, Capital Medical University, Beijing, China[3]Hematology Oncology Center, Beijing Children’s Hospital, Capital Medical University, Beijing, China[*1]Hematology Oncology Center, Beijing Children’s Hospital, Capital Medical University, 56 Nanlishi Road, West District, Beijing 100045, China.[*2]Department of Pharmacy, Beijing Children’s Hospital, Capital Medical University, 56 Nanlishi Road, West District, Beijing 100045, China.
推荐引用方式(GB/T 7714):
Cheng Xiaoling,Yan Kuo,Ma Jingyao,et al.Efficacy and safety of eltrombopag in the treatment of severe chronic immune thrombocytopenia in children of China: A single-center observational study[J].INTERNATIONAL JOURNAL OF IMMUNOPATHOLOGY AND PHARMACOLOGY.2019,33:-.doi:10.1177/2058738419872120.
APA:
Cheng, Xiaoling,Yan, Kuo,Ma, Jingyao,Chen, Zhenping,Zhao, Libo...&Wu, Runhui.(2019).Efficacy and safety of eltrombopag in the treatment of severe chronic immune thrombocytopenia in children of China: A single-center observational study.INTERNATIONAL JOURNAL OF IMMUNOPATHOLOGY AND PHARMACOLOGY,33,
MLA:
Cheng, Xiaoling,et al."Efficacy and safety of eltrombopag in the treatment of severe chronic immune thrombocytopenia in children of China: A single-center observational study".INTERNATIONAL JOURNAL OF IMMUNOPATHOLOGY AND PHARMACOLOGY 33.(2019):-