Efficacy and safety of avatrombopag in combination with standard immunosuppressive therapy for severe aplastic anemia

作者：Jianping Li, Weiru Liang, Huihui Fan, Kang Zhou, Yuan Li, Wenrui Yang, Liping Jing, Li Zhang, Lei Ye, Youzhen Xiong, Guangxin Peng, Yang Yang, Weiping Yuan, Jun Shi, Fengkui Zhang, Xin Zhao · 发表于：Experimental Hematology · 年份：2024 · DOI：10.1016/j.exphem.2024.104670 · 被引用次数：5 · 研究领域：Blood groups and transfusion、Platelet Disorders and Treatments、Hematopoietic Stem Cell Transplantation

Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disease. The addition of eltrombopag to immunosuppressive therapy (IST) improves the response rate, but its hepatotoxicity is concerning. Avatrombopag (AVA), a small-molecule thrombopoietin-receptor agonist without hepatotoxicity, has unknown efficacy in SAA treatment. This retrospective study assessed the efficacy and safety of AVA added to IST 42 SAA patients compared to a historical cohort of 84 patients receiving IST alone, using propensity score matching. The median age was 31.5 (6.0-67.0 years) years old in group A and 26 (16.0-45.0 years) years old in group B. At 3 months, group A showed higher complete response (CR) and overall response (OR) rates than group B (CR: 19.0% vs. 4.8%, p = 0.024; OR: 54.8% vs. 39.3%, p = 0.145). Higher CR and OR rates were also found at 6 months in group A than in group B (CR: 31.0% vs. 14.3%, p = 0.145; OR 71.4% vs. 51.2%, p = 0.048). In multivariate analysis of group A, a shorter interval from disease onset to antithymocyte globulin (ATG) treatment (≤6 months) (p = 0.005) predicted better responses rate at 6 months. Event-free survival was also improved in group A (60.7% vs. 49.6%). AVA was well-tolerated, with no hepatic injury observed during treatment, even in those with pre-existing hepatic impairment. The addition of AVA to IST improves both the response rate and response quality in patients with SAA while ensuring safety.