Hetrombopag plus porcine ATG and cyclosporine for the treatment of aplastic anaemia: early outcomes of a prospective pilot study

Experimental Hematology & Oncology

Correspondence

Youzhen Xiong¹  Jianping Li¹  Fengkui Zhang¹  Xin Zhao¹  Liping Jing¹  Xu Liu¹  Lei Ye¹  Wenrui Yang¹  Huihui Fan¹  Yang Yang¹  Kang Zhou¹  Yuan Li¹  Li Zhang¹  Guangxin Peng¹ 

[1] 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, 300020, Tianjin, China;

关键词: Hetrombopag;    Aplastic anaemia;    Immunosuppressive therapy;   

DOI  :  10.1186/s40164-023-00377-3

 received in 2022-11-14, accepted in 2023-01-27,  发布年份 2023

来源: Springer

【 摘 要 】

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.

【 授权许可】

CC BY   
© The Author(s) 2023

【 图 表 】

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