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- Title
Efficacy, Effectiveness, and Safety of Interventions for Von Hippel-Lindau Associated Renal Cell Carcinoma: A Systematic Literature Review.
- Authors
Jonasch, Eric; Balijepalli, Chakrapani; Yan, Kevin; Gullapalli, Lakshmi; Li, Joyce; Sundaram, Murali
- Abstract
Background: A small proportion of renal cell carcinoma (RCC) are associated with hereditary syndromes such as von Hippel-Lindau disease (VHL) and are commonly treated with surgical interventions. More recently, systemic treatments for VHL-associated RCC have been assessed as an alternative to surgery. Methods: A systematic literature review was conducted by searching MEDLINE, EMBASE, and Cochrane Registry of Controlled Trials to collect and interpret published evidence on treatments for VHL-associated RCC patients to better understand the treatment landscape. Results: This review identified 32 primary studies, comprised of single-arm clinical trials and real-world studies assessing systemic, surgical, radiological, or image guided ablation interventions. In clinical trials, treatment with sunitinib and pazopanib showed an objective response in 33% and 52% of RCC lesions respectively. For patients treated with belzutifan, 64% of patients showed an objective response, of which 7% were complete response and 57% were partial responses with a 24-month PFS rate of 96%. In real-world studies, treatment with sunitinib, pazopanib, axitinib, and sorafenib showed an objective response in 40%, 0%, 33%, and 25% of RCC lesions respectively, and all the responses were partial. In the studies assessing surgical, radiological, or image guided ablation interventions primary failure/re-intervention rates ranged from 2% to 84%. Conclusion: Local procedures are still a mainstay in the management of patients with non-metastatic VHL-associated RCC although multiple procedures incur an increasing rate of complications. A limited number of clinical trials and real-world studies evaluated VEGF-TKIs for the treatment of VHL-RCC, while responses were observed, long term treatment was limited by toxicities.
- Publication
Kidney Cancer (2468-4562), 2024, Vol 8, Issue 1, p1
- ISSN
2468-4562
- Publication type
Article
- DOI
10.3233/KCA-230021