Tobias Else, Eric Jonasch, Othon Lliopoulos, Kathryn E Beckermann, Vivek Narayan, Benjamin L Maughan, Stephane Oudard, Jodi K Maranchie, Ane B Iversen, Cynthia Muller Goldberg, Wei Fu, Rodolfo F Perini, Yanfang Liu, W Marston Linehan, Ramaprasad Srinivasan
PURPOSE: Primary analysis of the ongoing, single-arm, phase 2 LITESPARK-004 study (NCT03401788) showed clinically meaningful antitumor activity in von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC) and other neoplasms with belzutifan treatment. We describe results of belzutifan treatment for VHL disease-associated pancreatic lesions (pancreatic neuroendocrine tumors [pNETs] and serous cystadenomas). PATIENTS AND METHODS: Adults with VHL diagnosis based on germline VHL alteration, ≥1 measurable RCC tumor, no renal tumor >3 cm or other VHL neoplasm requiring immediate surgery, Eastern Cooperative Oncology Group performance status of 0 or 1, and no prior systemic anticancer treatment received belzutifan 120 mg once daily...
February 23, 2024: Clinical Cancer Research