Division of Cancer Care and Epidemiology, Queen's Cancer Research Institute, Kingston, Canada; Departments of Oncology, Queen's University, Kingston, Canada.
Division of Cancer Care and Epidemiology, Queen's Cancer Research Institute, Kingston, Canada.
J Cancer Policy. 2021 Dec;30:100301. doi: 10.1016/j.jcpo.2021.100301. Epub 2021 Aug 25.
Progression-free survival (PFS) is often used as a clinical trials outcome for evaluating new therapies for solid tumors. While PFS is a validated surrogate for overall survival (OS) or quality of life (QOL) in some settings, it is increasingly used in contexts where surrogacy is not established. PFS is a composite endpoint of survival, symptomatic progression, and imaging-only progression. The intrinsic value of asymptomatic (imaging-only) progression from the patient perspective is not known.
Patients with advanced metastatic cancer (lung, colorectal, or ovarian) participated in a discrete choice experiment, with a structured treatment choice trade-off exercise. The interview guide and visual aids were developed by a multidisciplinary team including patient representatives. Participants were provided with a hypothetical clinical scenario and treatment options resulting in the same OS duration. A sliding scale was used for duration of delay in imaging progression to determine each patient's willingness to trade longer time for a given level of toxicity.
20 (11 M, 9 F) patients participated. 85 % (n = 17) of patients chose treatment with less toxicity and shorter duration even if associated with a shorter time to worsening imaging. Two patients chose a trade-off for a more toxic treatment with an increase in imaging PFS by 18 months and 24 months respectively. One patient chose to always opt for most aggressive treatment irrespective of PFS benefit and toxicity.
Most patients with metastatic cancer currently being treated with palliative chemotherapy considered delayed imaging progression in the absence of OS gain to be of low value.
PFS should not be assumed to have intrinsic value to patients in the absence of surrogacy for OS or QOL when making drug treatment and policy decisions.
无进展生存期（PFS）通常被用作评估实体瘤新疗法的临床试验结果。虽然在某些情况下，PFS 是总生存期（OS）或生活质量（QOL）的验证替代指标，但它越来越多地用于替代指标尚未确立的情况下。PFS 是生存、症状进展和仅影像学进展的复合终点。从患者角度来看，无症状（仅影像学）进展的内在价值尚不清楚。
患有晚期转移性癌症（肺癌、结直肠癌或卵巢癌）的患者参与了离散选择实验，进行了结构化的治疗选择权衡练习。访谈指南和视觉辅助工具由包括患者代表在内的多学科团队开发。参与者提供了一个假设的临床情况和治疗选择，这些选择导致相同的 OS 持续时间。使用滑动比例来确定影像学进展的延迟时间，以确定每个患者愿意为给定水平的毒性换取更长时间的意愿。
20 名（11 名男性，9 名女性）患者参与了研究。85%（n=17）的患者选择了毒性较小、持续时间较短的治疗方法，即使这与影像学恶化的时间较短有关。两名患者选择了毒性更大的治疗方法进行权衡，分别增加了 18 个月和 24 个月的影像学 PFS。一名患者选择始终选择最积极的治疗方法，无论 PFS 获益和毒性如何。
目前正在接受姑息性化疗治疗的转移性癌症患者大多数认为，在没有 OS 获益的情况下，影像学进展延迟的价值较低。
在没有 OS 或 QOL 替代指标的情况下，不应假设 PFS 对患者具有内在价值，在做出药物治疗和政策决策时。
