Yang Bixi, Fu Lingling, Li Hongmin, Chen Hui, Zhang Rui, Yao Jiafeng, Zhang Liqiang, Wu Runhui, Ma Jie
Department of Hematology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Hematologic Disease Laboratory, Hematology Center, Beijing, China.
Pediatr Res. 2025 Apr;97(5):1590-1596. doi: 10.1038/s41390-024-03253-w. Epub 2024 May 31.
Severe aplastic anemia (SAA) is caused by immune-mediated destruction. Standard immunosuppressive therapy (IST) is effective but needs to be improved.
The data of patients with SAA and received IST were analyzed retrospectively to conducted this historical control study.
A total of 115 SAA patients (60 males; median age of 5.77 years and median follow-up time of 45 months) were enrolled in this study. The complete response rates (CRR) of the eltrombopag group at 3 and 6 months were higher than the control group (30.3% vs.8.2% at 3 months; 50.0% vs. 10.2% at 6 months). The overall response rates (ORR) showed no differences. There were significant differences in the times from G-CSF, Red blood cell transfusion, and Platelet transfusion between the two groups. No difference in overall survival (OS), event-free survival (EFS), and relapse rate between two groups. There is no variable were associated with prognosis in both groups.
Addition of eltrombopag to IST confers faster hematological response and higher early hematological response in pediatric SAA patients.
Addition of eltrombopag to standard immunosuppressive therapy confers faster hematological response and higher early hematological response in pediatric severe aplastic anemia patients. Eltrombopag showed reliable safety but had no impact on long-term response and prognosis. This article is a historical controlled study consisting of 115 pediatric severe aplastic anemia patients and makes up for the lack of clinical data deficient on pediatric severe aplastic anemia with TPO-RA combined with IST.
重型再生障碍性贫血（SAA）由免疫介导的破坏引起。标准免疫抑制治疗（IST）有效，但仍需改进。
回顾性分析接受IST的SAA患者数据以进行这项历史对照研究。
本研究共纳入115例SAA患者（60例男性；中位年龄5.77岁，中位随访时间45个月）。艾曲泊帕组在3个月和6个月时的完全缓解率（CRR）高于对照组（3个月时分别为30.3%和8.2%；6个月时分别为50.0%和10.2%）。总缓解率（ORR）无差异。两组在使用粒细胞集落刺激因子（G-CSF）、红细胞输注和血小板输注的时间上存在显著差异。两组在总生存期（OS）、无事件生存期（EFS）和复发率方面无差异。两组均无与预后相关的变量。
在IST基础上加用艾曲泊帕可使儿童SAA患者获得更快的血液学反应和更高的早期血液学反应。
在标准免疫抑制治疗中加用艾曲泊帕可使儿童重型再生障碍性贫血患者获得更快的血液学反应和更高的早期血液学反应。艾曲泊帕显示出可靠的安全性，但对长期反应和预后无影响。本文是一项由115例儿童重型再生障碍性贫血患者组成的历史对照研究，弥补了血小板生成素受体激动剂（TPO-RA）联合IST治疗儿童重型再生障碍性贫血临床数据的不足。
