(UroToday.com) The 2023 AUA annual meeting included an advanced kidney cancer session, featuring a presentation by Dr. Federico Belladelli discussing results from a prospective surveillance study assessing the generalizability of the MK-6482-004 trial to real-world VHL patients. The use of MK-6482 (Belzutifan) may completely change the treatment of VHL patients with clear cell RCC, potentially reducing unnecessary surgeries along with the consequent renal functional impairment.1 Since no data about the generalizability of the study to real-world VHL patients is available, the study aimed to investigate the generalizability of belzutifan using a prospective database of VHL patients enrolled in a multidisciplinary surveillance program.
Since January 2021, a multidisciplinary program devoted to VHL disease is operational at Dr. Belladelli’s institution (San Raffaele, Milan, IT). All consenting patients with a VHL diagnosis based on pathogenic variant were enrolled in a prospective, observational surveillance study. In the outpatient clinic, each patient received diagnostic and clinical evaluation by a multidisciplinary team of physicians to cover all the potential areas of interest in VHL disease. The primary outcome was the overall inclusion rate and the inclusion/exclusion criterion-specific rate of patients that would have access to the MK-6482-004 trial. Inclusion criteria were:
- Diagnosis of VHL pathogenic variant
- At least one measurable RCC ≥10 mm in diameter
- Eastern Cooperative Oncology Group performance-status [ECOG-PS] score of 0 or 1
Exclusion criteria were RCC requiring immediate surgery and metastatic disease.
Overall, 38 patients were enrolled in the VHL program. The median age was 46 (range: 19-70) and the median eGFR was 92 mL/min/1.73m2. The rate of real-world VHL patients that would have been included in the trial was 8% (n=3) only. Specifically:
- 63% (n=24) of the patients were excluded owing to absence of any measurable renal cell carcinoma ≥10 mm in diameter
- 37% (n=14) of patients were excluded owing to ECOG-PS being higher than 1
- 5% (n=2) of patients would have been excluded due to evidence of metastatic disease
Differences in descriptive statistics of baseline characteristics between MK-6482-004 patients and real-world patients are summarized as follows:
Dr. Belladelli concluded his presentation by discussing results from a prospective surveillance study assessing the generalizability of the MK-6482-004 trial to real-world VHL patients with the following take-home messages:
- Despite the enthusiasm generated by the MK-6482-004 trial, the findings of the study appear applicable to a small proportion of real world VHL patients
- Larger studies, focusing also on extra-renal VHL lesions are required to better define the role of MK-6482 (Belzutifan) for the treatment of VHL patients
Presented by: Federico Belladelli, MD, IRCCS, San Raffaele, Milan, Italy
Written by: Zachary Klaassen, MD, MSc – Urologic Oncologist, Assistant Professor of Urology, Georgia Cancer Center, Augusta University/Medical College of Georgia, @zklaassen_md on Twitter during the 2023 American Urological Association (AUA) Annual Meeting, Chicago, IL, April 27 – May 1, 2023
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