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：
Accounts of Chemical Research presents short, concise and critical articles offering easy-to-read overviews of basic research and applications in all areas of chemistry and biochemistry. These short reviews focus on research from the author’s own laboratory and are designed to teach the reader about a research project. In addition, Accounts of Chemical Research publishes commentaries that give an informed opinion on a current research problem. Special Issues online are devoted to a single topic of unusual activity and significance.
Accounts of Chemical Research replaces the traditional article abstract with an article "Conspectus." These entries synopsize the research affording the reader a closer look at the content and significance of an article. Through this provision of a more detailed description of the article contents, the Conspectus enhances the article's discoverability by search engines and the exposure for the research.
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