Belzutifan, a Potent HIF2α Inhibitor, in the Pacak–Zhuang Syndrome
Junne Kamihara, Kayla V. Hamilton, Jessica A. Pollard, Catherine M. Clinton, Jill A. Madden, Jasmine Lin, Alma Imamovic, Catherine B. Wall, Ari J. Wassner, Brent R. Weil, Matthew M. Heeney, Sara O. Vargas, William G. Kaelin, Katherine A. Janeway, Rodolfo F. Perini, Naseem J. Zojwalla, Stephan D. Voss, Steven G. DuBois
Abstract
. Treatment with belzutifan led to a rapid and sustained tumor response along with resolution of hypertension, headaches, and long-standing polycythemia. This case shows the application of a targeted therapy for the treatment of a patient with a rare tumor-predisposition syndrome. (Funded by the Morin Family Fund for Pediatric Cancer and Alex's Lemonade Stand Foundation.).
Topics & Concepts
MedicineCancerMutationGermline mutationGenetic testingBioinformaticsPatient careSomatic cellCancer treatmentOncologyMutation testingIntensive care medicineCancer researchClinical PracticeInternal medicineRare diseaseBlood cancerCancer, Hypoxia, and MetabolismAdrenal and Paraganglionic TumorsCardiac tumors and thrombi