Table 1.

Patient characteristics, indication for belzutifan initiation, dose adjustments, belzutifan treatment duration, and ocular response

Case	Age	Ocular disease	Systemic disease	VHL variation(s)	Prior ocular treatments	Indication	Initial dose	Side effects	Adjusted dose	Duration	Ocular response
1	Teens	>80 RHs	CNS	Gln73del2caGG	Laser, Cryo, EBR, PPV, and IVI	RH	80 mg daily	Fatigue and light-headedness	40 mg 5x/weekly	12 months	Involution*
2	40s	RH optic nerve OU and <10 peripheral RHs	PCC	Tyr98His TAC>CAC	Laser, EBR, IVI, and PDT	RH	120 mg daily	Anemia	80 mg daily	9 months	Involution
3	20s	RH optic nerve	pNET	Arg167Gln CGG>CAG	None	pNET	120 mg daily	Fatigue, anemia, and nausea	40 mg daily	13 months	Involution
4	20s	<10 RHs	CNS and pNET	Leu63 del4ctGCGC	Laser and Cryo	CNS	120 mg daily	None	None	19 months	No new tumors
5	50s	<10 RHs	CNS, pNET, and PCC	Val130Leu GTT>CTT	Laser	CNS	120 mg daily	Tinnitus, HA, and LE edema	80 mg daily	13 months	No new tumors
6	30s	>20 RHs	CNS and RCC	Asn78Ser AAT>AGT	Enucleation OD and laser	RCC	120 mg daily	Anemia	80 mg daily	24 months	No new tumors
7	50s	<10 RHs	RCC and CNS	Val130Leu GTT>CTT	Laser	RCC	120 mg daily	Anemia	80 mg daily	4 months	No new tumors

CNS, central nervous system hemangioblastoma; PCC, pheochromocytoma; pNET, pancreatic neuroendocrine tumor; RCC, renal cell carcinoma; RH, retinal hemangioblastoma; Cryo, cryotherapy; EBR, external beam radiation; PPV, pars plana vitrectomy; IVI, intravitreal injections of anti-vascular endothelial growth factor and steroids; PDT, photodynamic therapy; HA, headache; LE, lower extremity.

*In this case, the ocular tumors had involuted after the initial dose. The dose was reduced to 40 mg three times weekly on which recurrent disease activity was noted. Upon readjustment of dose to 40 mg five times weekly, involution was again noted.

Note: Ocular and systemic diseases refer to the presence of these findings prior to the initiation of belzutifan.