In this #UrologyJournalClub we discuss and summarize the Belzitufan Phase 2 trial for renal masses in patients with Von Hippel-Lindau (VHL) disease published @NEJM which led to the approval of Welireg.
Pts with VHL and renal masses (assumed to be renal cell carcinoma, biopsy not required).
Most pts had other VHL-related tumors simultaneously but these were not considered for inclusion or exclusion criteria but measured either way.
2/
This study found an objective response rate of 49% (quite impressive for a drug in an orphan disease), another 49% of patients showed stable disease and only 3 patients had progression.
"men with confirmed M1 castration-resistant prostate cancer (CRPC) whose disease had progressed during treatment with enza or abi, administered for M1 or M0 CRPC or M1 hormone-sensitive prostate cancer. Previous taxane chemotherapy was allowed.”
Now look at control arm 👀
2/8
So.. to be included u had to progress to the control arm, that way, chances of doing well, correct me if I’m wrong but are pretty low.
One may argue that you can give it either way, but to the best of my knowledge this is NOT standard of care.
3/8