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.
🔗 pubmed.ncbi.nlm.nih.gov/34818478/
💾urologiabe.files.wordpress.com/2021/11/urolog…
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@VPrasadMDMPH
🔗 pubmed.ncbi.nlm.nih.gov/34818478/
💾urologiabe.files.wordpress.com/2021/11/urolog…
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@VPrasadMDMPH
📃 phase 2, single arm, open label
N=61
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.
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N=61
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.
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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.
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It is relevant to mention these patients tipically will have multiple ablative procedures/surgeries during their lifetime to treat recurrent RCC and therefore diminishing the number of procedures and delaying the time between them is a clinically relevant outcome.
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Most patients in this trial had already undergone 4 or more procedures for RCC.
Something very positive about this drug was its large effect on other VHL-related tumours, making it kind of a magic bullet able to treat many tumors with the same drug and AE profile.
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Something very positive about this drug was its large effect on other VHL-related tumours, making it kind of a magic bullet able to treat many tumors with the same drug and AE profile.
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Surprisingly this data with such a short follow-up was enough for the FDA to approve it ( fda.gov/drugs/resource… ), and not only that, but extended the indication for ALL tumors associated with VHL even though these were not part of the protocol ( accessdata.fda.gov/drugsatfda_doc… )
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Without a control arm; if this drug becomes standard of care we'll never know how long does this drug delay ablative/surgical interventions (PFS or stable disease is not enough as there may be an artifact by indolent disease) nor its effect in quality of life.
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Would patients prefer the continuous AEs or the increased risk of needing interventions?
It will become impossible to have a conversation with patients about pros and cons.
I know that this is an orphan disease and that it is difficult to recruit such patients...
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It will become impossible to have a conversation with patients about pros and cons.
I know that this is an orphan disease and that it is difficult to recruit such patients...
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But if it was possible to do it for Everolimus in Tuberous Sclerosis (EXIST-2 trial) I think it could be done here as well ( pubmed.ncbi.nlm.nih.gov/23312829/ )
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Finally...
How much will Welireg cost? Aprox. 27,000 USD for one month of treatment.
Should healthcare systems cover this drug without a control arm?
What are your thoughts?
#UroSoMe
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How much will Welireg cost? Aprox. 27,000 USD for one month of treatment.
Should healthcare systems cover this drug without a control arm?
What are your thoughts?
#UroSoMe
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