Abstract

Eltrombopag is being investigated for the treatment of aplastic anemia (AA) by stimulating hematopoietic stem cell (HSC) proliferation. To evaluate the efficacy and safety of eltrombopag in the first-line therapy of pediatric AA. The present retrospective study assessed pediatric patients with newly diagnosed AA administered immunosuppressive therapy (IST) (rabbit ATG combined with CSA) with eltrombopag at a single center from March to September 2017. All patients were followed up for >2 years. A total of 14 patients (8 males), averagely aged 86 months, were enrolled in this study. Eltrombopag was administered with a median time to initiation of 19.5 days after IST; the median course of treatment was 253 days. Complete and overall response rates at 6 months were 64.3% (9/14 case) and 78.6% (11/14 cases), respectively. The survival rate was 100%, and no relapse occurred in responders. Eltrombopag was well-tolerated; however, the most common adverse events included indirect bilirubin elevation, jaundice, and transient liver-enzyme elevation. By the end of follow-up, bone marrow chromosomes were normal, and no abnormal myelodysplastic syndrome (MDS)-related clones appeared. Addition of eltrombopag to IST is associated with markedly increased complete response with respect to hematology in pediatric patients with SAA compared with a historical cohort, without intolerable side effects.

摘要

艾曲波帕正在研究通过刺激造血干细胞 (HSC) 增殖来治疗再生障碍性贫血 (AA)。评价艾曲波帕在小儿 AA 一线治疗中的疗效和安全性。本回顾性研究评估了 2017 年 3 月至 2017 年 9 月在单个中心接受艾曲波帕的新诊断 AA 免疫抑制治疗 (IST)（兔 ATG 联合 CSA）的儿科患者。所有患者均随访 > 2 年。本研究共招募了 14 名患者（8 名男性），平均年龄 86 个月。艾曲波帕在 IST 后 19.5 天开始给药的中位时间；中位疗程为253天。6 个月时的完全缓解率和总体缓解率分别为 64.3%（9/14 例）和 78.6%（11/14 例）。成活率为100%，响应者没有复发。艾曲波帕耐受性良好；然而，最常见的不良事件包括间接胆红素升高、黄疸和短暂的肝酶升高。随访结束时，骨髓染色体正常，未出现异常骨髓增生异常综合征（MDS）相关克隆。与历史队列相比，在 IST 中添加艾曲波帕与 SAA 儿科患者血液学的完全反应显着增加有关，并且没有无法忍受的副作用。未出现异常骨髓增生异常综合征（MDS）相关克隆。与历史队列相比，在 IST 中添加艾曲波帕与 SAA 儿科患者血液学的完全反应显着增加有关，并且没有无法忍受的副作用。未出现异常骨髓增生异常综合征（MDS）相关克隆。与历史队列相比，在 IST 中添加艾曲波帕与 SAA 儿科患者血液学的完全反应显着增加有关，并且没有无法忍受的副作用。