Endolymphatic Sac Tumors Associated With von Hippel-Lindau: A Case Report Highlighting Opportunity for Novel Orphan Drug Therapy.

Objective: To discuss the potential benefit of belzutifan therapy in a patient with von Hippel-Lindau (VHL) disease-associated endolymphatic sac tumor (ELST).
Patients: Case report.
Interventions: Clinical details of a patient with residual ELST after hearing preservation surgery who initiated belzutifan therapy postoperatively for concurrent renal cell carcinoma, as well as literature review of belzutifan and ELST.
Main Outcome Measures: The patient remained without radiologic evidence of growth of her residual tumor at 17 months post-initiation of belzutifan. It is unknown whether this represents therapeutic drug effect, nonviability of residual tumor, or slow tumor growth not captured radiographically within the duration of follow-up.
Conclusions: Belzutifan could have direct therapeutic benefit in patients with VHL-associated ELST.
Competing Interests: The authors otherwise have no relevant disclosures with regard to financial support, acknowledgement, or conflicts of interest.
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