Zhang Zhuxin, Hu Qinglin, Yang Chen, Chen Miao, Han Bing
Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing, China.
Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Shuaifuyuan No. 1, Dongcheng District, Beijing 100730, China.
Ther Adv Hematol. 2023 Sep 14;14:20406207231191310. doi: 10.1177/20406207231191310. eCollection 2023.
Eltrombopag (ELT), a thrombopoietin receptor agonist (TPO-RA), has been approved for relapsed/refractory aplastic anemia (AA). However, data on avatrombopag (AVA), another TPO-RA, are limited, and the comparisons between the two TPO-RAs are lacking.
We aimed to compare the efficacy and safety between ELT and AVA in relapsed/refractory AA patients.
In this retrospective study, patients with relapsed/refractory AA who had been treated with ELT ( = 45) or AVA ( = 30) alone and had compatible baseline hematological parameters were compared.
Data from patients diagnosed with acquired AA were retrospectively collected. All patients were refractory/relapsed to standard immunosuppressive therapy (IST) for at least 6 months before ELT or AVA. Patients had to be treated with ELT or AVA alone for at least 6 months before evaluation if they did not respond. Baseline characteristics, overall response (OR), complete response (CR), relapse, adverse events, and factors that may affect efficacy were analyzed.
Of the 75 patients enrolled, 45 received ELT and 30 received AVA. Patients with AVA had a higher percentage of abnormal liver or renal function than those with ELT ( 0.036). No significant difference was found in the OR/CR rate in the first/second/third/sixth month between the two cohorts ( 0.05). Patients treated with AVA had a shorter median time to response than those treated with ELT ( 0.012) and had a higher platelet level in the second month ( 0.041). AVA had fewer adverse events than ELT ( 0.046). Under compatible follow-up time ( 0.463), no difference was found between the ELT and AVA cohorts in relapse ( 1.000) or clone evolution ( 0.637). No predictive factors for OR and CR in the sixth month were found for either ELT or AVA.
With worse liver or renal function, AVA had a similar OR/CR rate but a shorter median time to response and fewer adverse events for patients with relapsed/refractory AA.
艾曲泊帕（ELT）是一种血小板生成素受体激动剂（TPO-RA），已被批准用于治疗复发/难治性再生障碍性贫血（AA）。然而，关于另一种TPO-RA阿伐曲泊帕（AVA）的数据有限，且缺乏两种TPO-RA之间的比较。
我们旨在比较ELT和AVA在复发/难治性AA患者中的疗效和安全性。
在这项回顾性研究中，比较了单独接受ELT（n = 45）或AVA（n = 30）治疗且基线血液学参数匹配的复发/难治性AA患者。
回顾性收集诊断为获得性AA患者的数据。所有患者在接受ELT或AVA治疗前至少6个月对标准免疫抑制治疗（IST）难治/复发。如果患者无反应，在评估前必须单独接受ELT或AVA治疗至少6个月。分析基线特征、总体缓解（OR）、完全缓解（CR）、复发、不良事件以及可能影响疗效的因素。
在纳入的75例患者中，45例接受ELT治疗，30例接受AVA治疗。AVA患者肝功能或肾功能异常的百分比高于ELT患者（P = 0.036）。两组在第1/2/3/6个月的OR/CR率无显著差异（P>0.05）。接受AVA治疗的患者中位缓解时间比接受ELT治疗的患者短（P = 0.012），且在第2个月血小板水平更高（P = 0.041）。AVA的不良事件比ELT少（P = 0.046）。在随访时间匹配的情况下（P = 0.463），ELT组和AVA组在复发（P = 1.000）或克隆演变方面无差异（P = 0.637）。未发现ELT或AVA在第6个月时OR和CR的预测因素。
对于复发/难治性AA患者而言，AVA在肝功能或肾功能较差的情况下，OR/CR率相似，但中位缓解时间较短，不良事件较少。
