Belzutifan for Renal Cell Carcinoma in von Hippel–Lindau Disease

The cause of von Hippel–Lindau disease is excess activity of the HIF-2α pathway. A total of 61 patients with renal cell carcinoma and other proliferative manifestations of the hypoxia-induced signaling pathway received belzutifan, which inhibits the HIF-2α pathway. Nearly half the patients with rena...

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Veröffentlicht in:	The New England journal of medicine 2021-11, Vol.385 (22), p.2036-2046
Hauptverfasser:	Jonasch, Eric, Donskov, Frede, Iliopoulos, Othon, Rathmell, W. Kimryn, Narayan, Vivek K, Maughan, Benjamin L, Oudard, Stephane, Else, Tobias, Maranchie, Jodi K, Welsh, Sarah J, Thamake, Sanjay , Park, Eric K, Perini, Rodolfo F, Linehan, W. Marston, Srinivasan, Ramaprasad
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Adverse events Age of Onset Aged Anemia - chemically induced Antineoplastic Agents - adverse effects Antineoplastic Agents - therapeutic use Basic Helix-Loop-Helix Transcription Factors - antagonists & inhibitors Cancer Carcinoma, Renal Cell - drug therapy Carcinoma, Renal Cell - etiology Central nervous system Disease Progression Fatigue - chemically induced Female Fentanyl Follow-Up Studies Gene expression Genetics Hemangioblastoma - drug therapy Hematology Humans Hypoxia Hypoxia-inducible factors Indenes - adverse effects Indenes - therapeutic use Kidney cancer Kidney Neoplasms - drug therapy Kidney Neoplasms - etiology Magnetic resonance imaging Male Metastasis Middle Aged Neoplasms, Multiple Primary - drug therapy Nephrology Nephrology General Nervous system Neuroendocrine tumors Neuroendocrine Tumors - drug therapy Oncology Oral administration Pancreatic Neoplasms - drug therapy Patients Renal cell carcinoma Solid tumors Statistical analysis Transcription activation Treatments in Oncology Vascular endothelial growth factor VHL protein Von Hippel-Lindau disease von Hippel-Lindau Disease - complications von Hippel-Lindau Disease - genetics
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Zusammenfassung:	The cause of von Hippel–Lindau disease is excess activity of the HIF-2α pathway. A total of 61 patients with renal cell carcinoma and other proliferative manifestations of the hypoxia-induced signaling pathway received belzutifan, which inhibits the HIF-2α pathway. Nearly half the patients with renal cell carcinoma had a response to treatment, and 98% had disease control.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa2103425