Development-free survival (PFS) is a surrogate endpoint is outlined as follows (by ChatGPT):
In essence, PFS refers back to the size of time throughout a medical examine or remedy routine the place a affected person’s illness doesn’t worsen or progress. As an example, in most cancers analysis, it measures the time from the beginning of remedy till the most cancers exhibits indicators of development, unfold, or relapse.Understanding PFS is significant as a result of it helps clinicians and researchers assess the effectiveness of a remedy in controlling the illness. An extended PFS means that the remedy is efficiently slowing down the development of the sickness. This metric is especially essential in medical trials when evaluating completely different remedy approaches or medicine.
A key query is, do sufferers care about PFS? The reply could also be sure, as a result of higher PFS is most frequently (however not at all times) correlated with longer survival. Nevertheless, do sufferers worth PFS unbiased of OS? Maybe so if their high quality of life is best (i.e., they’ve fewer signs) in the course of the pre-progression section. Or, maybe they’ve decrease anxiousness in the event that they know their most cancers has not progressed.
What does the literature say?
A paper by Raphael et al. (2019) performed a scientific literature evaluation of research that consider whether or not sufferers with superior most cancers perceive and worth PFS. Total, 17 research met their inclusion standards. Of those:
Ten research particularly offered sufferers with the time period progression-free survival as an attribute alternative. Within the phrases used to outline the attribute of PFS, 6 research used the time period survival. 5 research clarified that PFS could not translate into higher total survival, and 5 research defined that enhancements in PFS could not mirror how nicely the affected person could really feel. No examine clarified {that a} PFS occasion may characterize both development or dying, and no examine outlined for the affected person what constituted development. The research assessed herein underrepresented ethnic and racial minorities (imply proportion of white sufferers, 88%; vary, 77%-96%). Values and preferences could range throughout cultural backgrounds on condition that completely different relative preferences had been assigned to price and efficacy outcomes in North American vs Asian research, though just a few research had been evaluated.
As you possibly can see from the desk under, there may be important heterogeneity when it comes to how PFS was offered to the most cancers affected person respondents.
In a number of the research, PFS was a very powerful attribute. In others, high quality of life elements had been extra essential. Total, nevertheless, it’s clear that extra analysis is required to completely perceive how sufferers understand the worth of PFS.
