Jin Yuanyuan, Li Ruixin, Lin Shengyun, Jia Jinsong, Yang Yan, Zhang Donghua, He Guangsheng, Li Jiangyong
Department of Hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, Collaborative Innovation Center for Cancer Personalized Medicine, 300 Guangzhou Road, Nanjing, Jiangsu, People's Republic of China.
Department of Hematology, Zhejiang Province Hospital of TCM, The First Affiliated Hospital of Zhejiang TCM University, Hangzhou, China.
Ann Hematol. 2022 Nov;101(11):2413-2419. doi: 10.1007/s00277-022-04966-w. Epub 2022 Aug 27.
Eltrombopag (EPAG), a thrombopoietin receptor agonist, was approved for the treatment of severe aplastic anemia (SAA) combined with immunosuppressive therapy (IST). However, the effects of real-life use of low doses of EPAG combined with rabbit antithymocyte globulin (ATG)-based IST in Asian patients with SAA are yet unknown. A total of 121 previously untreated Chinese patients with SAA were enrolled in a multicenter registry of the Chinese Eastern Collaboration Group of Anemia (2014-2020): 67 patients received IST alone and 54 patients received additional EPAG. Patients receiving IST plus EPAG had a higher overall response rate (ORR) at 1 month (P = 0.002), 3 months (P = 0.028), 6 months (P = 0.006), and 12 months (P = 0.031) compared to those receiving IST alone. EPAG was the favorable factor for response efficacy at 6 months. The complete response rate in the EPAG plus IST group was 17% at 3 months, 27% at 6 months, and 32% at 12 months, compared to 7% (P = 0.069), 14% (P = 0.11), and 33% (P = 0.92) for those treated with IST alone. The 2-year overall survival rate in EPAG plus IST and IST alone groups was 98% and 88%, respectively (P = 0.078). The rate of adverse events, including clonal evolution, infection, and transaminitis, was similar in the two cohorts. The addition of EPAG to IST was well-tolerated and associated with high rates of hematologic responses among the previously untreated Chinese patients with SAA.
艾曲泊帕（EPAG）是一种血小板生成素受体激动剂，已被批准用于联合免疫抑制治疗（IST）治疗重型再生障碍性贫血（SAA）。然而，在亚洲SAA患者中，低剂量EPAG联合基于兔抗胸腺细胞球蛋白（ATG）的IST在实际应用中的效果尚不清楚。共有121例既往未接受治疗的中国SAA患者纳入了中国东部贫血协作组多中心登记研究（2014 - 2020年）：67例患者仅接受IST治疗，54例患者额外接受EPAG治疗。与仅接受IST治疗的患者相比，接受IST加EPAG治疗的患者在1个月（P = 0.002）、3个月（P = 0.028）、6个月（P = 0.006）和12个月（P = 0.031）时的总缓解率（ORR）更高。EPAG是6个月时反应疗效的有利因素。EPAG加IST组在3个月时的完全缓解率为17%，6个月时为27%，12个月时为32%，而仅接受IST治疗的患者分别为7%（P = 0.069）、14%（P = 0.11）和33%（P = 0.92）。EPAG加IST组和仅IST组的2年总生存率分别为98%和88%（P = 0.078）。两个队列中不良事件的发生率，包括克隆演变、感染和转氨酶升高，相似。在既往未接受治疗的中国SAA患者中，IST联合EPAG耐受性良好，且血液学反应率高。
