骨髓增生异常综合症
医学
临床试验
低甲基化剂
重症监护医学
癸他滨
肿瘤科
内科学
骨髓
生物化学
基因
基因表达
化学
DNA甲基化
作者
Guillermo Garcia‐Manero
标识
DOI:10.1016/s2352-3026(22)00265-4
摘要
Single-agent hypomethylating agents remain the cornerstone of treatment for patients with high-risk myelodysplastic syndromes. Although these agents have clinical activity and can improve the overall survival of these patients, their impact on the natural history of myelodysplastic syndromes is only partial. Therefore, we need either newer agents or combinations that could have a greater impact on the survival of our patients. Over the past decade there has been an increased effort in drug development for myelodysplastic syndromes. Hypomethylating agent combinations that have been explored over the past decade include agents that block mutant TP53, NEDD inhibitors, BCL-2 inhibitors, and antibodies such as sabatolimab or magrolimab. Despite initial encouraging results, two registration trials from 2021 and 2022 have not been successful in improving outcomes when compared with single-agent hypomethylating agents. Here, I summarise the current status of ongoing phase 3 trials for patients with untreated high-risk myelodysplastic syndromes and provide some suggestions for future designs.
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