Yang Wenrui, Zhao Xin, Liu Xu, Xiong Youzhen, Fan Huihui, Zhang Li, Li Jianping, Ye Lei, Zhou Kang, Li Yuan, Yang Yang, Peng Guangxin, Jing Liping, Zhang Fengkui
State Key Laboratory of Experimental Hematology, Haihe Laboratory of Cell Ecosystem, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, 288 Nanjing Road, Heping District, Tianjin, 300020, China.
Exp Hematol Oncol. 2023 Feb 1;12(1):16. doi: 10.1186/s40164-023-00377-3.
Hetrombopag, a small molecular thrombopoietin-receptor agonist, has shown encouraging efficiency in immunosuppressive therapy refractory or relapsed severe aplastic anaemia. To investigate the response rate of hetrombopag combined with IST as first-line treatment, we designed a prospective pilot study including 32 patients with SAA treated with anti-human T lymphocyte porcine immunoglobulin (p-ATG), cyclosporine, and hetrombopag. In addition, 96 patients with SAA treated with p-ATG and cyclosporine alone were matched as controls. In total, 21.9% of patients treated with hetrombopag achieved complete response (CR) at 3 months, while 5.2% of patients achieved CR in the control group (P = 0.005). At 6 months, the CR rates were 34.4% in the hetrombopag group and 14.6% in the control group (P = 0.015). The overall response rates at 6 months were 68.7% and 50.0% in the hetrombopag and control groups, respectively. The median time to haematologic response was 56 days and 77 days, and to CR was 96 days and 214 days in the hetrombopag and control groups, respectively. In conclusion, adding hetrombopag to IST as first-line treatment resulted in faster and better haematologic response in SAA.
艾曲泊帕是一种小分子血小板生成素受体激动剂，已在免疫抑制治疗难治或复发的重型再生障碍性贫血中显示出令人鼓舞的疗效。为了研究艾曲泊帕联合免疫抑制治疗（IST）作为一线治疗的缓解率，我们设计了一项前瞻性试点研究，纳入32例接受抗人T淋巴细胞猪免疫球蛋白（p-ATG）、环孢素和艾曲泊帕治疗的重型再生障碍性贫血患者。此外，将96例仅接受p-ATG和环孢素治疗的重型再生障碍性贫血患者作为对照。总体而言，接受艾曲泊帕治疗的患者中有21.9%在3个月时达到完全缓解（CR），而对照组中这一比例为5.2%（P = 0.005）。在6个月时，艾曲泊帕组的CR率为34.4%，对照组为14.6%（P = 0.015）。6个月时，艾曲泊帕组和对照组的总缓解率分别为68.7%和50.0%。艾曲泊帕组和对照组血液学缓解的中位时间分别为56天和77天，达到CR的中位时间分别为96天和214天。总之，在IST基础上加用艾曲泊帕作为一线治疗可使重型再生障碍性贫血患者获得更快、更好的血液学缓解。
