MP24-02: Survey based study of emerging management strategies for VHL

Neal Patel*, Aileen Arvelo, Los Angeles, CA, Gennady Bratslavsky, Syracuse, NY, Chandra Clark, Boston, MA, Othon Iliopoulos, Charlestown, MA, Eric Jonasch, Houston, TX, Sandy Liu, Los Angeles, CA, Joshua Mann, Boston, MA, Peter Muraki, Brian Shuch, Los Angeles, CA

Introduction: Belzutifan was recently FDA approved for the management of Von Hippel-Lindau syndrome (VHL).  Given the disease complexity and morbidity of recurrent surgery, a well-tolerated systemic agent has been long-awaited. Balancing benefit with chronic usage and toxicity must be considered. To gain insight in future usage, a survey was provided to VHL kidney cancer experts in the United States.

Methods: A survey was developed by members of the VHL Family Alliance Advisory Council. The survey was distributed to practitioners involved in the care of VHL kidney cancer patients at designated VHL Alliance Care Centers and National Comprehensive Cancer Network centers. Center contacts were asked to distribute the survey amongst kidney cancer specialists who would be involved in VHL kidney cancer care. Surveys were anonymous and hosted on a secure, web-based platform. VHL practitioners were asked about the feasibility of using belzutifan, which specialty should administer, clinical indications for renal and non-renal manifestations, and optimal duration of therapy. Descriptive statistics were performed and Pearson chi-square analyses to evaluate differences between groups.

Results: A total of 59 respondents from 30 institutions participated. Urologists (51%) and medical oncologists (42%) represented the majority of participants. The majority (98%) of respondents anticipated that belzutifan’s approval would significantly change the current kidney cancer treatment landscape and therapy should be continuous(76%). 43% of urologists planned to prescribe belzitufan compared to 84% of medical oncologists(p=0.006). Only 9% of medical oncologists believed that urologists should be involved in belzutifan administration as opposed to 38% of urologist(p=0.015). In individuals with renal tumors <3 cm with low anticipated surgical morbidity, only 36% would continue the prior treatment paradigm of surveillance then surgery while 36% would initiate belzutifan to prevent growth. In those with multifocal disease with growth of a solitary lesion while on belzutifan, 50% would proceed with isolated treatment of that site.

Conclusions: Most VHL kidney cancer specialists anticipate a paradigm shift in the field with the approval of belzutifan. Provider roles may change with movement away from local treatment. Opinions on which specialists should administer belzutifan differ by specialty.  Treatment indications on topics such as when to initiate therapy and how to best salvage vary widely. Collaborative efforts amongst experts to accumulate additional clinical data will help guide a new era of VHL management.

Source of Funding: None