Key result
Belzutifan treatment in a patient with Pacak-Zhuang syndrome led to a rapid and sustained tumor response and the resolution of hypertension, headaches, and long-standing polycythemia.
Why the study?
Does belzutifan improve tumor response and symptoms in a patient with Pacak-Zhuang syndrome?
Does belzutifan improve tumor response and symptoms in a patient with Pacak-Zhuang syndrome?
Belzutifan may be an effective targeted therapy for patients with Pacak-Zhuang syndrome, leading to tumor response and symptom resolution.
May allow medical control of secondary hypertension and polycythemia; leaves open generalizability beyond this single case.
. 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.).
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A 2021 study studied this question. Belzutifan treatment in a patient with Pacak-Zhuang syndrome led to a rapid and sustained tumor response and the resolution of hypertension, headaches, and long-standing polycythemia.
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