Hetrombopag Plus Porcine ATG and Cyclosporine for the Treatment of Aplastic Anaemia: Early Outcomes of a Prospective Pilot Study

Authors

Wenrui Yang
Xin Zhao
Xu Liu
Youzhen Xiong
Huihui Fan
Li Zhang
Jianping Li
Lei Ye
Kang Zhou
Yuan Li
Yang Yang
Guangxin Peng
Liping Jing
Fengkui Zhang

Publication Date

2-1-2023

Journal

Experimental Hematology & Oncology

Abstract

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.

Keywords

Hetrombopag, Aplastic anaemia, Immunosuppressive therapy

Comments

Supplementary Materials

PMID: 36726169