Table 2.
Literature review of studies describing belzutifan use in patients with VHL-associated RHs
| Study | Patients | Age | Sex | Indication | Dose, mg | Adjusted dose, mg | Dose adjusted for side effects? | Ocular tumor response | Follow-up on drug, months |
|---|---|---|---|---|---|---|---|---|---|
| Cotton et al. [9] (2023) | 1 | 71 | F | RH | 120 | 80 | Anemia | Reduction in tumor size | 12 |
| Ercanbrack et al. [10] (2024) | 3 | 23 | M | CNS and pancreas | 120 | NR | Discontinued due to anemia | Tumor shrunk and then lasered when medication held | 21 |
| 32 | M | CNS | 120 | 80 | Anemia and fatigue | Tumors decreased in size | 5 | ||
| 44 | M | CNS | 120 | 40 | Shortness of breath and anemia | Tumor OD decreased to 50% size with a reduction in exudative detachment | 7 | ||
| Fairbanks et al. [11] (2023) | 1 | 24 | M | RH | NR | NR | NR | Reduction in size and number of tumors | 6 |
| Grimes et al. [12] (2023) | 2 | 40 | M | CNS | 120 | N/A | No | Decreased size and reduction in vascularity and exudation | 1 |
| 66 | F | Renal | 120 | NR | NR | Decreased in size and fluid | 30 | ||
| Jonasch et al. [6] (2021) | 12* | NR | NR | Renal | 120 | NR | NR | 100% showed improvement | Median 21.8 (entire study) |
| Jones et al. [13] (2023) | 1 | 15 | M | RH | 120 | N/A | No | Regression in tumor size and vascularity | 12 |
| Mustafi et al. [14] (2023) | 1 | 11 | F | RH | 120 | 80 | Anemia | Decreased size and decreased feeding/draining vessels and exudative detachment | 6 |
NR, not reported; N/A, not applicable; RH, retinal hemangioblastoma.
*Study for use in renal cell carcinoma, 12 patients with ocular tumors.