与 von Hippel-Lindau 相关的内淋巴囊肿瘤：病例报告彰显新型孤儿药治疗的机会。

IF 1.9 3区医学 Q3 CLINICAL NEUROLOGY Otology & Neurotology Pub Date : 2024-10-01 Epub Date: 2024-08-21 DOI:10.1097/MAO.0000000000004307

Donald Tan, Rance J T Fujiwara, Christopher Tan, Brandon Isaacson, Jacob B Hunter

{"title":"与 von Hippel-Lindau 相关的内淋巴囊肿瘤：病例报告彰显新型孤儿药治疗的机会。","authors":"Donald Tan, Rance J T Fujiwara, Christopher Tan, Brandon Isaacson, Jacob B Hunter","doi":"10.1097/MAO.0000000000004307","DOIUrl":null,"url":null,"abstract":"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.","PeriodicalId":19732,"journal":{"name":"Otology & Neurotology","volume":null,"pages":null},"PeriodicalIF":1.9000,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Endolymphatic Sac Tumors Associated With von Hippel-Lindau: A Case Report Highlighting Opportunity for Novel Orphan Drug Therapy.\",\"authors\":\"Donald Tan, Rance J T Fujiwara, Christopher Tan, Brandon Isaacson, Jacob B Hunter\",\"doi\":\"10.1097/MAO.0000000000004307\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"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.\",\"PeriodicalId\":19732,\"journal\":{\"name\":\"Otology & Neurotology\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":1.9000,\"publicationDate\":\"2024-10-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Otology & Neurotology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1097/MAO.0000000000004307\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/8/21 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q3\",\"JCRName\":\"CLINICAL NEUROLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Otology & Neurotology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1097/MAO.0000000000004307","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/8/21 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}

引用次数: 0

摘要

摘要讨论贝珠替凡治疗冯-希佩尔-林道（VHL）病相关内淋巴囊肿瘤（ELST）患者的潜在益处：病例报告：一名听力保留手术后残留ELST的患者因同时患有肾细胞癌而在术后开始接受贝珠替凡治疗的临床细节，以及有关贝珠替凡和ELST的文献综述：主要结果指标：患者在接受贝珠替凡治疗17个月后，其残留肿瘤仍无放射学生长迹象。目前尚不清楚这是否代表药物治疗效果、残留肿瘤无法存活，还是在随访期间未通过放射学检查发现肿瘤缓慢生长：结论：贝祖替凡对VHL相关ELST患者有直接的治疗效果。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

查看原文

微信好友朋友圈 QQ好友复制链接

本刊更多论文

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.

求助全文

通过发布文献求助，成功后即可免费获取论文全文。去求助

来源期刊

Otology & Neurotology 医学-耳鼻喉科学

CiteScore

3.80

自引率

14.30%

发文量

509

审稿时长

3-6 weeks

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.