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 scientific examine or remedy routine the place a affected person’s illness doesn’t worsen or progress. For example, in most cancers analysis, it measures the time from the beginning of remedy till the most cancers reveals indicators of development, unfold, or relapse.
Understanding PFS is important 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 vital in scientific trials when evaluating totally 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 impartial of OS? Maybe so if their high quality of life is healthier (i.e., they’ve fewer signs) through the pre-progression part. Or, maybe they’ve decrease nervousness in the event that they know their most cancers has not progressed.
What does the literature say?
A paper by Raphael et al. (2019) carried out a scientific literature assessment of research that consider whether or not sufferers with superior most cancers perceive and worth PFS. General, 17 research met their inclusion standards. Of those:
Ten research particularly introduced sufferers with the time period progression-free survival as an attribute selection. 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 replicate how effectively the affected person could really feel. No examine clarified {that a} PFS occasion might symbolize 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 share of white sufferers, 88%; vary, 77%-96%). Values and preferences could range throughout cultural backgrounds on condition that totally different relative preferences have been assigned to value and efficacy outcomes in North American vs Asian research, though just a few research have been evaluated.
As you possibly can see from the desk beneath, there’s important heterogeneity when it comes to how PFS was introduced to the most cancers affected person respondents.
In a few of the research, PFS was a very powerful attribute. In others, high quality of life components have been extra vital. General, nevertheless, it’s clear that extra analysis is required to completely perceive how sufferers understand the worth of PFS.
