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The 2022 Annual Meeting of the European Society of Oncology (ESMO) was held
Hipper-Lindau syndrome (VHL) is a rare autosomal dominant disorder manifesting as angioblastoma involving the cerebellum, spinal cord, kidneys, and retina
background
The transcription factor HIF-2α is a recognized carcinogenic driver whose activation in VHL disease is caused
method
Adult patients who are eligible (germ cell VHL mutation, non-metastatic RCC measurable lesion ≥1, RCC lesion without >3 cm requiring immediate surgery, previous systemic antineoplastic therapy, ECOG PS 0-1) are treated with belzutifan (120 mg once daily) until disease progression, intolerable toxicity occurs, or the patient withdraws consent
Figure 1 Study design
outcome
As of 1 April 2022, 38/61 (62%) of patients were still receiving treatment, and the main reasons for discontinuation of treatment were patient selection (n=11,18%) and disease progression (n=6,10%)
Figure 2 Remission in patients with RCC
The incidence of grade 3 treatment-related adverse events (TRAEs) was 18% (n=11), with the most common being anemia (n=7,11%)
.
No level 4 or 5 TRAEs occur
.
In the case of a median follow-up period of 37.
8 months, belzutifan showed good antitumor activity and controllable safety against VHL-associated RCCs
.
Reference Sources
LR
Reviewed: LR
Execution: LR