慢性疾病中的白内障和浪费:监管和临床试验更新
Francesco Fioretti1,2, Stephan von Haehling3,4, Andrew J S Coats5,6
1Baylor Scott & White Research Institute, Dallas, Texas, USA.
Journal of cachexia, sarcopenia and muscle
|November 27, 2025
概括
有效的癌症缓解症疗法仍然有限,目前的治疗方法显示功能益处不一致. 未来的试验必须优先考虑以患者为中心的结果和经过验证的措施,以获得监管机构的批准和改善患者的生活质量.
科学领域:
- 在瘤学瘤学.
- 代谢过程中的代谢.
- 临床试验 临床试验
背景情况:
- 癌症缓解症显著影响患者的生活质量,治疗耐受性和生存率,影响50%-80%的癌症患者.
- 目前的治疗策略,包括营养支持和药理学药物,产生有限和不一致的功能益处.
- 在临床试验中测量肌肉质量的现有方法,如CT扫描,由于可变性和缺乏临床相关性,作为主要终点是不充分的.
研究的目的:
- 总结 Society on Cachexia and Wasting Disorders (SCWD) 关于监管要求和卡切西亚临床试验进展的研讨会的主要讨论.
- 突出挑战和在临床试验终点上的不断变化的共识,以缓疗法.
- 审查最近的临床试验数据和新兴的治疗候选人来管理癌症缓解症.
主要方法:
- 来自SCWD监管和试验更新工作坊的讨论摘要.
- 审查现有的缓解症治疗方法及其有效性.
- 对提交的临床试验数据 (MENAC,TCMCB07,ART27.13,S-pindolol) 的分析以及对试验终点的监管观点.
主要成果:
- 现有的缓冲症治疗方法显示出混合的结果,往往无法实现持续的功能改善.
- 监管机构强调患者报告的结果,身体功能和发病率-死亡率作为关键的终点,超越孤立的身体组成措施.
- 像TCMCB07和ART27.13这样的新兴药物在稳定体重和改善热量摄入方面表现有希望,而S-pindolol的目标是肌肉质量和力量.
结论:
- 有效的缓解症疗法需要转向以患者为中心的目标和验证的终点,这些终点反映了现实生活中的功能.
- 临床试验设计必须整合客观的功能测量和患者报告的结果,以满足监管标准并证明有意义的临床益处.
- 未来的研究和开发应该集中在新的治疗机制和多模式干预措施上,以解决癌症缓存症的复杂性质.
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