Belzutifan in a Patient With VHL-Associated Metastatic Pancreatic Neuroendocrine Tumor.
J Natl Compr Canc Netw
; 20(12): 1285-1287, 2022 12.
Article
em En
| MEDLINE
| ID: mdl-36509068
ABSTRACT
von Hippel-Lindau (VHL) disease is a rare autosomal-dominant hereditary disease characterized by mutation of the VHL gene. This gene encodes for the VHL protein, which regulates the activity of HIF-α, a transcription factor involved in the cellular response to hypoxia. Mutations in VHL lead to the accumulation of HIF-α and, consequently, the engagement of hypoxia-sensitive genes with tumorigenic effects. VHL disease is associated with the development of tumors in multiple organs, including pancreatic neuroendocrine tumors (pNETs). Belzutifan is an HIF-α inhibitor; however, it has not been previously evaluated in patients with metastatic or treatment-refractory pNETs. This report presents a 43-year-old woman with VHL-associated metastatic pNET treated with belzutifan after progression on multiple systemic therapies. She began treatment with belzutifan and experienced partial radiographic response within 1 month of treatment. Other than asymptomatic anemia, no adverse effects developed during 5 months of ongoing therapy. Belzutifan is an inhibitor of HIF-2α that targets the underlying pathophysiology of VHL-associated pNETs. Our case report describes exceptional activity in a metastatic pNET arising from VHL.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Neoplasias Pancreáticas
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Carcinoma de Células Renais
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Tumores Neuroendócrinos
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Doença de von Hippel-Lindau
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Neoplasias Renais
Tipo de estudo:
Risk_factors_studies
Limite:
Adult
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Female
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Humans
Idioma:
En
Revista:
J Natl Compr Canc Netw
Assunto da revista:
NEOPLASIAS
Ano de publicação:
2022
Tipo de documento:
Article