Understanding the Impact of Belzutifan on Treatment Strategies for Patients with VHL
Aileen Arevalo1, Neal Patel2,3, Peter Muraki3
1David Geffen School of Medicine, University of California, Los Angeles, CA, USA.
Abstract:
Belzutifan was recently approved for the management of Von Hippel-Lindau disease (VHL). Given the morbidity of recurrent treatment, systemic therapy to reduce or eliminate the need for surgery has been long-awaited. Herein, we sought to gain insight about future utilization by surveying VHL kidney cancer experts in the United States. A survey developed by members of the VHL Alliance (VHLA) Clinical Advisory Council was distributed to kidney cancer providers at VHLA and National Comprehensive Cancer Network (NCCN) centers. Surveys were administered on a secure web-based platform. A total of 60 respondents from 29 institutions participated. Urologists (50%) and medical oncologists (43%) represented the majority of participants. The majority (98%) of respondents anticipated that belzutifan's approval would significantly change the current treatment landscape. Most reported that therapy should be continuous (76%). There was a difference in willingness to prescribe belzutifan by specialty (38% of urologists vs 91% of medical oncologists (P = 0.02)). In individuals with renal tumors <3 cm, 36% would still recommend surveillance, while 36% would initiate belzutifan to prevent growth. In those with multifocal renal lesions and growth of a solitary tumor on belzutifan, 50% would proceed with only treatment of that site. In conclusion, VHL kidney cancer specialists anticipate a paradigm shift with the approval of belzutifan. Provider roles may change with movement away from surgical management. Opinions on treatment indications, such as when to initiate therapy and how to best salvage, vary widely and therefore collaborative efforts among experts may assist in the development of new clinical guidelines.
Insights
Belzutifan
Area of Science:
- Oncology
- Genitourinary Cancer
- Vascular Endothelial Growth Factor Inhibitors
Background:
- Von Hippel-Lindau (VHL) disease is a genetic disorder associated with increased risk of various cancers, particularly renal cell carcinoma.
- Recurrent surgical interventions for VHL-associated tumors lead to significant morbidity.
- Belzutifan, a novel HIF-2α inhibitor, offers a systemic treatment option for VHL disease.
Purpose of the Study:
- To survey kidney cancer experts in the United States regarding the anticipated utilization of belzutifan for Von Hippel-Lindau disease.
- To understand expert opinions on treatment indications, initiation, and management strategies with belzutifan.
Main Methods:
- A survey was developed by the VHL Alliance (VHLA) Clinical Advisory Council.
- The survey was distributed to kidney cancer providers at VHLA and National Comprehensive Cancer Network (NCCN) centers.
- Data was collected from 60 respondents (urologists and medical oncologists) across 29 institutions via a secure web-based platform.
Main Results:
- 98% of experts anticipate belzutifan will significantly alter the VHL treatment landscape.
- 76% of respondents favor continuous therapy with belzutifan.
- Significant differences exist in prescribing intent between urologists (38%) and medical oncologists (91%) (P=0.02).
Conclusions:
- VHL kidney cancer specialists foresee a paradigm shift with belzutifan approval, potentially reducing reliance on surgical management.
- Expert opinions on initiating belzutifan therapy and managing treatment variations are diverse, highlighting the need for guideline development.
- Collaborative efforts among experts are crucial for establishing standardized clinical guidelines for belzutifan use in VHL disease.
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