晚期癌症患者无进展生存期的价值。

The value of progression-free survival to patients with advanced-stage cancer.

机构信息

Sussex Health Outcomes Research & Education in Cancer, Brighton and Sussex Medical School, University of Sussex, Falmer, East Sussex BN1 9RX, UK. l.j.fallowfield@ sussex.ac.uk

出版信息

Nat Rev Clin Oncol. 2011 Oct 18;9(1):41-7. doi: 10.1038/nrclinonc.2011.156.

Abstract

Progression-free survival (PFS) is frequently used as a primary end point in oncology clinical trials. Employing PFS instead of overall survival as the primary outcome has the advantage that trial completion can be quicker with fewer patients required, and it is cheaper. PFS is sensitive to cytostatic as well as cytotoxic mechanisms of therapeutic intervention and directly measures the effect of the investigational treatment. Despite these practical advantages, it is unclear whether or not extending PFS provides discernable clinical benefit. New treatments that increase PFS may not be of sufficient value to patients with advanced-stage cancer unless accompanied by tangible quantity or quality of life advantages. Any symptom relief that patients gain from treatment resulting in tumor shrinkage or stabilization must be balanced against the toxic effects that drug therapy itself creates. Consequently, improved assessment of new treatments using patient-reported outcomes alongside PFS is crucial to enable communication between clinicians and patients and optimal decision-making about therapeutic options.

摘要

无进展生存期(PFS)常被用作肿瘤临床试验的主要终点。与总生存期相比,使用 PFS 作为主要结局有以下优势:所需患者更少,试验完成更快,且成本更低。PFS 对细胞抑制和细胞毒性治疗干预机制均敏感,可直接测量研究治疗的效果。尽管具有这些实际优势,但 PFS 的延长是否能提供明显的临床获益尚不清楚。对于晚期癌症患者,延长 PFS 的新治疗方法可能没有足够的价值,除非伴随着生活质量的明显改善。任何因治疗导致肿瘤缩小或稳定而使患者获得的症状缓解,都必须与药物治疗本身带来的毒副作用相平衡。因此,使用患者报告的结果与 PFS 一起,对新治疗方法进行改进评估至关重要,这有助于临床医生和患者之间的沟通,并对治疗选择做出最佳决策。

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