期刊
BRITISH JOURNAL OF HAEMATOLOGY
卷 196, 期 2, 页码 329-335出版社
WILEY
DOI: 10.1111/bjh.17892
关键词
myelodysplastic syndromes; clinical trials; drug development
类别
Our study found that most patients with myelodysplastic syndromes (MDS) require therapeutic intervention, but there is a lack of approved treatments. Success rates in MDS trials are low, taking longer to complete compared to average clinical trials, with many trials failing to adequately evaluate important factors like quality of life.
Most patients with myelodysplastic syndromes (MDS) require therapeutic intervention. However, there are few approved treatments for MDS. To explore reasons, we searched clinicaltrials.gov and clinicaltrialsregister.eu for MDS trials from 2000 to 2020. We assessed which agents were under investigation and analysed clinical trial characteristics and continuation rates from phase I to II to III to approval. As such, we identified 384 unique agents in 426 phase I, 430 phase II and 48 phase III trials. Success rates for phase III trials and agents were low, and MDS trials took markedly longer to complete than the average clinical trial. Although success rates were higher when MDS-specific phase I trials were conducted, 52% of the agents had not been evaluated in a phase I trial for MDS. MDS trials often failed to include quality of life, an especially important outcome for older MDS patients. Our work identifies factors potentially contributing to the paucity of available agents for MDS. We suggest a framework to improve clinical research in MDS that might ultimately augment the number of available agents.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据