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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Belzutifan Group | Subjects received Belzutifan |
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| Measure | Description | Time Frame |
|---|---|---|
| Objective response rate | RECIST 1.1 | Month 6 and Month 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Radiographic response | By RECIST 1.1 | 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 |
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Inclusion Criteria:
Exclusion Criteria:
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Adult patients diagnosed with VHL syndrome who choose to use belzutifan for their treatment
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| David Ka-Wai LEUNG, FRCS, MBChB | Contact | 35053933 | davidleung@surgery.cuhk.edu.hk |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Prince of Wales Hospital | Shatin | Hong Kong |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34268287 | Background | Zhou T, Guan H, Wang L, Zhang Y, Rui M, Ma A. Health-Related Quality of Life in Patients With Different Diseases Measured With the EQ-5D-5L: A Systematic Review. Front Public Health. 2021 Jun 29;9:675523. doi: 10.3389/fpubh.2021.675523. eCollection 2021. | |
| 40274483 | Background | Linehan WM, Pinto CA, Liu Y, Choo K, Gautam R, Fox C, Roy A, Li M, Bosan R, Nielsen D, Ryan B, Blake Z, Reynolds K, Rompre-Brodeur A, Pinto PA, Vocke C, Gurram S, Ball MW, Perini R, Srinivasan R. Longitudinal Evaluation of Clear-cell Renal Cell Carcinoma in von Hippel-Lindau Disease. Eur Urol. 2025 Jul;88(1):56-63. doi: 10.1016/j.eururo.2025.03.002. Epub 2025 Apr 23. |
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| ID | Term |
|---|---|
| D006623 | von Hippel-Lindau Disease |
| ID | Term |
|---|---|
| D020752 | Neurocutaneous Syndromes |
| D009422 | Nervous System Diseases |
| D000798 | Angiomatosis |
| D014652 | Vascular Diseases |
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| Baseline, Month 6 and Month 12 |
| Health-economic impact of belzutifan | By per-patient emergence department and inpatient cost | Month 6, Month 12, Month,18, Month 24, Month 30, Month 36 |
| 35709961 | Background | Louise M Binderup M, Smerdel M, Borgwadt L, Beck Nielsen SS, Madsen MG, Moller HU, Kiilgaard JF, Friis-Hansen L, Harbud V, Cortnum S, Owen H, Gimsing S, Friis Juhl HA, Munthe S, Geilswijk M, Rasmussen AK, Moldrup U, Graumann O, Donskov F, Gronbaek H, Stausbol-Gron B, Schaffalitzky de Muckadell O, Knigge U, Dam G, Wadt KA, Bogeskov L, Bagi P, Lund L, Stochholm K, Ousager LB, Sunde L. von Hippel-Lindau disease: Updated guideline for diagnosis and surveillance. Eur J Med Genet. 2022 Aug;65(8):104538. doi: 10.1016/j.ejmg.2022.104538. Epub 2022 Jun 13. |
| 34818478 | Background | Jonasch E, Donskov F, Iliopoulos O, Rathmell WK, Narayan VK, Maughan BL, Oudard S, Else T, Maranchie JK, Welsh SJ, Thamake S, Park EK, Perini RF, Linehan WM, Srinivasan R; MK-6482-004 Investigators. Belzutifan for Renal Cell Carcinoma in von Hippel-Lindau Disease. N Engl J Med. 2021 Nov 25;385(22):2036-2046. doi: 10.1056/NEJMoa2103425. |
| D002318 |
| Cardiovascular Diseases |
| D000072661 | Ciliopathies |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |