对近期耐火性或复发性骨髓瘤的II期试验进行系统审查:我们能否为未来的试验设计提供信息?
Roelof van Ewijk1, Morgane Cleirec2, Nikolas Herold3
1Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.
Cancer treatment reviews
|September 22, 2023
概括
最近的骨髓瘤II期试验显示,有针对性的治疗研究的增加,但试验设计的持续异质性. 需要进一步优化,以改善对这种具有挑战性的癌症的新治疗方法的开发.
科学领域:
- 在瘤学瘤学.
- 临床试验设计 临床试验设计
- 药理学研究 药理学研究
背景情况:
- 复发性/耐药性骨髓瘤的预后不佳,需要改进临床试验策略.
- 之前对II期试验 (2003-2016) 的分析为评估最近的趋势提供了基准.
研究的目的:
- 分析2017年至2022年期间复发性/耐药性骨髓瘤II期临床试验设计的变化.
- 确定治疗策略,患者选择和终点的趋势,以告知未来的试验开发.
主要方法:
- 在试验登记册中注册的II期试验的系统审查 (01/01/2017-14/02/2022).
- 与2003-2016年间发现的98项试验进行了比较分析.
- 对这两个时期的出版物搜索和分析.
主要成果:
- 确定了越来越多的试验,有针对性的治疗评估的显著增加 (66%与41%相比).
- 单疗仍然是主要的治疗方法;然而,在患者纳入标准,研究设计和终点方面,异质性仍然存在.
- 生物标志物驱动的试验占总数的25%,82%包括儿科或青少年患者. 效率数据在很大程度上仍然令人失望.
结论:
- 尽管试验数量增加和新疗法的研究增加,但在II期骨髓瘤试验设计中仍然存在显著的异质性.
- 通过更深入的生物学理解来优化试验设计,对于推动骨髓瘤的新疗法开发至关重要.
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