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NEJM: Belzutifan is effective in treating VHL-related tumors

 Last Update: 2021-12-04
 Source: Internet
 Author: User

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plural form carcinoma

pellagra disease

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Every Heber - Lindau syndrome (VHL) is a rare familial disease, the main lesions are congenital vascular neuroblastoma (hemangioblastomas), it is a virulence gene VHL caused
.

There is 1 VHL patient in every 27,300-39,000 newborns, and its clinical manifestations are clear cell renal cell carcinoma, pancreatic neuroendocrine tumors, central nervous system and retinal hemangioblastomas

Vascular Belzutifan is a HIF-2α inhibitor.

This study is a phase II clinical study.
Patients with VHL-related renal cell carcinoma received 120 mg Belzutifan orally once a day to evaluate its safety and effectiveness
.

The primary endpoint of the study is the objective response rate, including complete or partial response (solid tumor treatment response evaluation criteria, version 1.

After an average follow-up of 21.
8 months, the overall objective response rate for renal cell carcinoma patients was 49%, in pancreatic cancer (47 of 61 patients, 77%) and central nervous system hemangioblastoma (15 of 50 patients) , 30%) also observed treatment response
.

All 16 eyes of 12 patients with retinal hemangioblastoma showed clinical improvement

The overall objective response rate for renal cell carcinoma patients was 49%, which was also observed in pancreatic cancer (47 of 61 patients, 77%) and central nervous system hemangioblastoma (15 of 50 patients, 30%) Arrived the treatment response

Patient response rate after Belzutifan treatment

Response rate of patients after treatment with Belzutifan Response rate of patients after treatment with Belzutifan

Studies believe that HIF-2α inhibitors have a good therapeutic effect on patients with Heber-Lindau syndrome-related tumors, and their adverse events are mostly grade 1-2 events
.

HIF-2α inhibitors have a good therapeutic effect on patients with Heber-Lindau syndrome-related tumors, and the adverse events are mostly grade 1-2 events
.

HIF-2α inhibitors have a good therapeutic effect on patients with Heber-Lindau syndrome-related tumors, and the adverse events are mostly grade 1-2 events

Original source:

Eric Jonasch et al.

Belzutifan for Renal Cell Carcinoma in von Hippel-Lindau Disease.
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