Donald Tan, Rance J T Fujiwara, Christopher Tan, Brandon Isaacson, Jacob B Hunter
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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.
期刊介绍：
Otology & Neurotology publishes original articles relating to both clinical and basic science aspects of otology, neurotology, and cranial base surgery. As the foremost journal in its field, it has become the favored place for publishing the best of new science relating to the human ear and its diseases. The broadly international character of its contributing authors, editorial board, and readership provides the Journal its decidedly global perspective.
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