Table 1.

Systemic and topical treatments for cutaneous neurofibromas.

	Drug	Mechanism of Action	Study Population	Results	Side Effects	Status
Systemic Treatments
	Imatinib mesylate 400 mg daily [18]	C-kit receptor tyrosine kinase inhibitor	1 adult patient	Small reduction in visual burden	Fluid retention, gastrointestinal symptoms, hepatotoxicity, fatigue, rash	N/A a
	Everolimus 5–15 ng/mL serum concentration [17]	mTOR b inhibitor	17 adult patients	Significant reduction in absolute value of paired lesion height and significant reduction in lesion surface volume in 3/17 patients	Gastrointestinal symptoms, upper respiratory infections, skin irritation	Complete (NCT02332902)
	Selumetinib c [23]	MEK d 1/2 inhibitor	Adult patients	N/A	N/A	Active, not recruiting (NCT02839720)
Topical Treatments
	Imiquimod 5% [24,25]	Toll-like receptor 7 agonist	11 adult patients	No significant reduction in tumor volume	Skin irritation and erythema	Complete (NCT00865644)
	Diphen-cyprone 0.04% [28]	Hapten that induces a delayed-type hypersensitivity reacion	Adult patients	N/A	Skin irritation and erythema	Active and recruiting (NCT05438290)
	NFX-179 0.05%, 0.15%, 0.50% [26]	MEK inhibitor	47 adult patients (35 in the treatment arms)	−1.6, −11.9, and −16.7, percent changes in CN volume for 0.05%, 0.15%, and 0.50% concentrations, respectively	Pruritis, stinging, erythema	Complete (NCT04435665)
	NFX-179 0.5%, 1.5% [27]	MEK inhibitor	Adult patients	N/A	N/A	Active, not recruiting (NCT05005845)

^a This study was a case report. ^b Mammalian target of rapamycin. ^c Dose not specified. ^d Mitogen-activated protein kinase.