Jain Shivi, Gernsheimer Terry, Kolodny Scott, Bernheisel Chelsea, Vredenburg Michael, Panch Sandhya R
Division of Hematology, Oncology, and Cell Therapy, Rush University Medical Center, Chicago, IL, USA.
Division of Hematology, University of Washington, Seattle, WA, USA.
Platelets. 2023 Dec;34(1):2195016. doi: 10.1080/09537104.2023.2195016.
Avatrombopag is an oral thrombopoietin receptor agonist (TPO-RA) that was approved in the US in 2019 for treatment of chronic immune thrombocytopenia (ITP). This post hoc analysis of the pivotal phase III study (NCT01438840) of avatrombopag in adult patients with ITP evaluated platelet count response to avatrombopag during the core study in different subgroups, and durability of response data in patients who responded to avatrombopag treatment both during the core phase (total population) and during the core and extension phase (total population and by subgroup). Loss of response (LOR [platelet count <30 × 10/L]) was defined as LOR over two consecutive scheduled visits. The response was generally similar between subgroups though a few differences were observed. The durability of response analysis showed that avatrombopag-treated patients maintained their response for 84.5% of time on treatment during the core phase and 83.3% during the core and extension phase; 55.2% of patients in the core phase and 52.3% in the core and extension phase never experienced LOR. We conclude that the initial response to avatrombopag is both stable and durable.
阿伐曲泊帕是一种口服血小板生成素受体激动剂（TPO-RA），于2019年在美国获批用于治疗慢性免疫性血小板减少症（ITP）。这项对阿伐曲泊帕治疗成年ITP患者的关键III期研究（NCT01438840）的事后分析，评估了在核心研究期间不同亚组中阿伐曲泊帕的血小板计数反应，以及在核心阶段（总体人群）和核心及扩展阶段（总体人群和各亚组）对阿伐曲泊帕治疗有反应的患者的反应持续性数据。反应丧失（LOR [血小板计数<30×10⁹/L]）定义为连续两次预定访视时出现LOR。各亚组之间的反应总体相似，不过也观察到了一些差异。反应持续性分析表明，在核心阶段，接受阿伐曲泊帕治疗的患者在治疗期间84.5%的时间保持反应，在核心及扩展阶段为83.3%；在核心阶段，55.2%的患者以及在核心及扩展阶段52.3%的患者从未经历过反应丧失。我们得出结论，对阿伐曲泊帕的初始反应既稳定又持久。
