Clark, as you will be aware, even the FDA doesn’t consider a comparator in this group to be ethical, so we can only compare to historical survival outcomes and with there being no current options in this population, the findings are notable.
Furthermore, why were they “aggressively funnelled to HSCT treatment.”
Could it be because they were seeing greater responses than as they would otherwise without Bex? TP53 patients are among the hardest to bridge… Centres won’t take uncontrolled TP53 disease to HSCT because outcomes are extremely poor.
So if anything, transplant is downstream of efficacy and supports outsized responses, not a source of bias…
As for “selection bias in specialist centres” that applies equally to every competing dataset. Yet agents like Magrolimab or Venetoclax haven’t shown comparable survival in the same setting, despite similar trial environments.