Abstract
Background Patients with von Hippel-Lindau syndrome (VHL) have an increased risk of developing multiple neoplasms. Recently, multiple pharmacologic interventions have been assessed for the treatment of these VHL-associated neoplasms. Objectives To identify current clinical trials evaluating pharmacological interventions in VHL-associated neoplasms, with an emphasis in renal cell carcinoma (RCC). Methods We conducted a systematic review of the literature utilising MEDLINE/PubMed and EMBASE databases. We searched for Clinical Trials in VHL and RCC to understand the current landscape of therapeutic interventions in this population. Results We identified five single-arms clinical trials assessing systemic interventions in patients with VHL and RCC. These therapeutic interventions consisted of three tyrosine kinase inhibitors (TKIs) – semaxinib, sunitinib, and pazopanib - and one hypoxia-inducible factor (HIF) inhibitor -belzutifan. Belzutifan therapy was associated with an overall response rate (ORR) of 49% in patients with VHL and RCC. Only 3% of patients experienced disease progression while on belzutifan, which resulted in an impressive 97% clinical benefit rate. Pazopanib was also associated with an ORR of 64%; no patients experienced disease progression while on therapy. Lastly, two studies investigated the role of sunitinib in patients with VHL and RCC. In these studies, sunitinib was associated with an ORR ranging from 33% to 66%. Conclusions Anti-angiogenic interventions such as TKI and HIF inhibitors have been shown to be effective in decreasing the rate of progression of VHL-associated neoplasms. Although only a few trials have evaluated different pharmaceutical interventions in VHL-associated neoplasms, understanding the molecular basis of this pathology has opened the opportunity for novel therapeutic approaches to improve outcomes in this population.
Published Version
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call