Efficacy and safety of lusutrombopag monotherapy for cyclosporine A-refractory, transfusion-dependent non-severe aplastic anemia
10.3760/cma.j.cn121090-20250225-00094
- VernacularTitle:芦曲泊帕治疗环孢素A难治性输血依赖型非重型再生障碍性贫血的疗效与安全性
- Author:
Yongxin ZHOU
1
;
Yangyang WEI
;
Ziwei LIU
;
Chen YANG
;
Miao CHEN
;
Bing HAN
Author Information
1. 中国医学科学院北京协和医院血液科，北京 100730
- Publication Type:Journal Article
- From:
Chinese Journal of Hematology
2025;46(10):963-966
- CountryChina
- Language:Chinese
-
Abstract:
This study retrospectively analyzed 12 patients with transfusion-dependent, non-severe aplastic anemia (TD-NSAA) refractory to cyclosporine A (CsA) , who were treated with lusutrombopag monotherapy. These patients either had a variety of chronic comorbidities or medication-related risks, or they were unresponsive to or intolerant of other thrombopoietin receptor agonists (TPO-RA) . The median treatment duration with lusutrombopag was 4 months (range: 3-11 months) , while the median follow-up period was 8 months (range: 6-11 months) . The overall response (OR) rates at months 3, 6, and the end of follow-up were 50.0%, 58.3%, and 50.0%, respectively, with a median time to OR of 2 months (range: 1-4 months) . Complete response (CR) rates were 8.3%, 16.7%, and 16.7% at the same time points, with a median time to CR of 4 months (range: 2-5 months) . Adverse events were all Grade 1, with an incidence rate of 25.0%. During follow-up, one patient experienced a loss of OR after discontinuing treatment, with a relapse rate of 14.3%; no clonal evolution or mortality was observed. These findings suggest that lusutrombopag is both effective and well-tolerated in CsA-refractory TD-NSAA patients and represents a promising therapeutic option for those with poor treatment tolerability.
