All other plots we use in oncology
Tell you what happens AFTER you start the protocol
A swimmers plot shows when treatment was given, and when response and progression occurred for each individual
A spider plot shows the tumor size for each patient, every time they were assessed, over time.
And a waterfall plot shows you the single best subsequent scan for each assessable patient.
All these scans have issues
If you select patients with very indolent biology, a swimmers plot will not distinguish drug activity from v. slow tumor growth
A spider plot is messy and hard to compare
A waterfall plot is the single best subsequent scan, but RECIST 1.1., uses a confirmatory scan for response, and thus, even 100% shrinkage is not always CR, and 30% is not always PR as it is fleeting
Previously Sunny Kim (OHSU fellow on job market)
Showed waterfall plots exaggerated effect size by ~12% b/c of the aforementioned reason as well as b/c it omits people who are too sick to be assessed
Our new paper in @EJCI_News argues that Randomized trials are necessary in medicine & PH for interventions w putative benefit & at best MED to LG effect size.
Parachutes & smoking are not good counter examples
Some people argue that b/c we did not need RCTs to know smoking is harmful or Parachutes are life saving, we don't need them to test cloth masking, or the Impella, or some new cancer drug, or HCQ, or <insert ur favorite practice>
But this is based on misunderstanding
There is a huge range of things we can do to someone that might hurt them or save them, imagine the spectrum (below)
Now out in @EJCI_News
Logan Powell & I show where randomized trials necessary
When people say 'we don't need an RCT of smoking (to prove harm) or parachutes (to prove benefit)' does that apply to widespread medical interventions?
🧵 onlinelibrary.wiley.com/doi/10.1111/ec…
Led by Timothee Olivier, our new paper is now out at @JAMANetworkOpen
We analyze 12 years of FDA approvals, and do the hard work of sorting them into
New Mechanism of Action (MOA)
& New MOA for that tumor type
Vs next in class
Few prelim thoughts on this trial (from quick read) #ASH21 1. It is not a 'second line' trial, it is a trial in the worst subset of second line pts & cannot extrapolate beyond
Primary refractory & relapse <12 mo
(TBH, a lot of people doing this already)
As such, it should not generalize to relapse > 12 months
2. That said, for those included, axi-cel seems preferable to chemo then auto; I am not surprised this is true in the most chemo insensitive biology. But a few more thoughts
3. This is Wrong, you are not supposed to do this 👇👇
Standard practice is to take these pts to CAR-T if needed in the control arm; thus, you must compare routine, upfront CAR-T to using CAR-T as salvage when indicated and standard of care.