无症状进展生存率作为多发性骨髓瘤中新兴的以患者为中心的终点:对MagnetsiMM-3试验数据的二次分析
Martin Kortüm1, Sebastian Theurich2, James Farrell3
1Würzburg University Hospital, Oberdürrbacherstr.6, 97080, Würzburg, Germany. Kortuem_M@ukw.de.
BMC cancer
|August 8, 2025
概括
一个新的无症状进展生存 (SPFS) 终点结合了临床进展和患者报告的结果,以更好地了解多发性骨髓瘤 (MM) 患者的经验.
科学领域:
- 血液学 血液学 血液学
- 在瘤学瘤学.
- 临床试验方法论 临床试验方法论
背景情况:
- 多发性骨髓瘤 (MM) 患者的经验需要超出临床进展的终点.
- 整合患者报告结果 (PROs) 与临床终点对于整体观点至关重要.
研究的目的:
- 开发和验证MM的新型复合终点,其中包括患者报告的症状.
- 评估患者报告的症状对MM患者疾病进展和存活率的影响.
主要方法:
- 系统的文献审查以识别MM症状,由专家Delphi小组改进.
- 对MagnetisMM-3试验数据的后期分析.
- 症状无进展生存期 (SPFS) 的发展,将无进展生存期 (PFS) 与关键PROs的恶化相结合.
主要成果:
- 发现了16种MM症状;与进展相关的昏昏欲睡,疲劳,疼痛和运动不良.
- 疼痛,疲劳或移动不良的恶化显著增加了进展风险,分别为22%,20%和31%.
- 没有达到中位数的SPFS,这表明试验群体的持续时间较长.
结论:
- 无症状进展生存率 (SPFS) 是整合PFS和PROs的MM中的第一个复合终点.
- SPFS提供了一个以患者为中心,全面衡量MM的治疗有效性和疾病影响的方法.
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