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In our study we also wanted to assess the toxic effects at the population level, which is a more relevant endpoint in risk assessment.
Nevertheless, good neurological outcome at hospital discharge is reasonably the more relevant endpoint.
Long term stabilization, such as lack of progression at 6 months may be a more relevant endpoint.
These findings, when combined with the overall results of this study, highlight the controversy regarding microbiologic versus clinical cure as the more relevant endpoint in patients with MDR-GN pneumonia.
In this case, ERα functional studies would be a more relevant endpoint to address this hypothesis in light of previous studies that demonstrate that BRCA1 interacts directly with ERα suppressing ERα-mediated transcription of target genes [ 25- 27].
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A more relevant pharmacodynamic endpoint in future may be to measure DNA strand breaks in tumour cells.
Being aware of this limitation, we further analyzed survival and the proportion of patients with good neurological outcome at hospital discharge as the more relevant primary endpoint.
While the results of this study had encouraged us to screen our patient population for these polymorphisms, when utilizing a more relevant clinical endpoint (symptomatic pulmonary embolism or deep venous thrombosis) we were unable to confirm this association.
This hindered comparison of progression-free survival (PFS) from the two evaluation methods and PFS would in this phase II trial have been a more relevant secondary endpoint than OS.
A functional outcome measure at discharge may be a more clinically relevant endpoint.
For example, in a field synopsis of pharmacogenetic studies, there was evidence of bias in which positive findings were more likely when examining surrogate markers of treatment effects rather than the more clinically relevant endpoints such as a disease complication or death.
More suggestions(16)
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