Comparison of eltrombopag and avatrombopag in the treatment of refractory/relapsed aplastic anemia: a single-center retrospective study in China.

IF 3.4 3区 医学 Q2 HEMATOLOGY Therapeutic Advances in Hematology Pub Date : 2023-09-14 eCollection Date: 2023-01-01 DOI:10.1177/20406207231191310
Zhuxin Zhang, Qinglin Hu, Chen Yang, Miao Chen, Bing Han
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Abstract

Background: 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.

Objectives: We aimed to compare the efficacy and safety between ELT and AVA in relapsed/refractory AA patients.

Design: In this retrospective study, patients with relapsed/refractory AA who had been treated with ELT (N = 45) or AVA (N = 30) alone and had compatible baseline hematological parameters were compared.

Methods: 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.

Results: 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 (p = 0.036). No significant difference was found in the OR/CR rate in the first/second/third/sixth month between the two cohorts (p > 0.05). Patients treated with AVA had a shorter median time to response than those treated with ELT (p = 0.012) and had a higher platelet level in the second month (p = 0.041). AVA had fewer adverse events than ELT (p = 0.046). Under compatible follow-up time (p = 0.463), no difference was found between the ELT and AVA cohorts in relapse (p = 1.000) or clone evolution (p = 0.637). No predictive factors for OR and CR in the sixth month were found for either ELT or AVA.

Conclusion: 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.

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艾曲波帕与阿伐曲波帕治疗难治性/复发性再生障碍性贫血的比较:一项在中国进行的单中心回顾性研究。
背景:Eltrombopag(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性/难治性再生障碍性贫血(AA)。然而,另一种TPO-RA--阿伐曲波帕(AVA)的数据有限,而且缺乏两种TPO-RA之间的比较:我们旨在比较 ELT 和 AVA 对复发/难治 AA 患者的疗效和安全性:在这项回顾性研究中,我们比较了接受ELT(45例)或AVA(30例)单独治疗的复发性/难治性AA患者,这些患者的基线血液学指标均符合要求:回顾性收集被诊断为获得性AA患者的数据。所有患者在接受ELT或AVA治疗前均已接受至少6个月的标准免疫抑制疗法(IST)治疗,且治疗无效或复发。如果患者没有应答,则必须在接受 ELT 或 AVA 治疗至少 6 个月后再进行评估。对基线特征、总体反应(OR)、完全反应(CR)、复发、不良事件以及可能影响疗效的因素进行了分析:在入组的75名患者中,45人接受了ELT治疗,30人接受了AVA治疗。AVA患者肝肾功能异常的比例高于ELT患者(P = 0.036)。两组患者在第一个月/第二个月/第三个月/第六个月的 OR/CR 率无明显差异(P > 0.05)。接受 AVA 治疗的患者比接受 ELT 治疗的患者获得应答的中位时间更短(p = 0.012),第二个月的血小板水平更高(p = 0.041)。与 ELT 相比,AVA 的不良反应较少(p = 0.046)。在相容的随访时间内(p = 0.463),ELT 和 AVA 组别在复发(p = 1.000)或克隆演变(p = 0.637)方面没有差异。ELT和AVA均未发现第6个月OR和CR的预测因素:结论:对于肝功能或肾功能较差的复发/难治性 AA 患者,AVA 的 OR/CR 率相似,但中位反应时间更短,不良反应更少。
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来源期刊
CiteScore
4.30
自引率
0.00%
发文量
54
审稿时长
7 weeks
期刊介绍: Therapeutic Advances in Hematology delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of hematology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in hematology, providing a forum in print and online for publishing the highest quality articles in this area.
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