This study aims to evaluate the Belzutifan's efficacy, drug safety, disease course, and the health-economic impact of the treatment to Von Hippel-Lindau (VHL) syndrome.
Adult patients diagnosed with VHL syndrome who choose to use belzutifan for their treatment will be identified and invited to participate in this registry. Clinical data, laboratory tests, imaging results and QOLs will be collected from subjects at baseline, every 3 months for the first year, then every 6 months until month 36 or cessation of the medication, whichever occurs first.
Study Type
OBSERVATIONAL
Enrollment
100
Prince of Wales Hospital
Shatin, Hong Kong
Objective response rate
RECIST 1.1
Time frame: Month 6 and Month 12
Radiographic response
By RECIST 1.1
Time frame: Month 6 and Month 12, Month 18, Month 24, Month 30 and Month 36
Change in Quality of Life score
By EQ-5D-5L. Score range from 0-100.The higher the score the better quality of life
Time frame: Baseline, Month 6 and Month 12
Health-economic impact of belzutifan
By per-patient emergence department and inpatient cost
Time frame: Month 6, Month 12, Month,18, Month 24, Month 30, Month 36
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