医学
耐受性
红细胞生成
不利影响
骨髓增生异常综合症
羟基脲
骨痛
贫血
骨髓纤维化
内科学
临床试验
输血
无效红细胞生成
骨髓
化疗
作者
Connie Kang,Yahiya Y. Syed
出处
期刊:Drugs
[Springer Nature]
日期:2021-05-10
卷期号:81 (8): 945-952
标识
DOI:10.1007/s40265-021-01527-9
摘要
Luspatercept (Reblozyl®), a first-in-class erythroid maturation agent, is approved in several countries worldwide for the treatment of adults with transfusion-dependent anaemia due to myelodysplastic syndromes (MDS), who have failed prior erythropoiesis-stimulating therapy, or β-thalassaemia. In pivotal, placebo-controlled, phase III trials, subcutaneous luspatercept significantly reduced red blood cell (RBC) transfusion requirements in patients with MDS or β-thalassaemia. Luspatercept had a generally manageable tolerability profile in clinical trials. Adverse events of special interest include thromboembolic events, hypertension and bone pain. Thus, luspatercept is an emerging treatment option in adults with transfusion-dependent anaemia due to MDS or β-thalassaemia.
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