Neurofibromatosis Type 1 Medication

Updated: Aug 03, 2018
  • Author: David T Hsieh, MD, FAAP; Chief Editor: Amy Kao, MD  more...
  • Print

Medication Summary

No known medical therapies are beneficial to patients with NF1. Several drug trials have been initiated, looking for medications that slow or halt the growth of neurofibromas. Thus far, none of these medications have demonstrated significant benefit, although various research trials involving chemotherapeutic and other agents are underway in an attempt to slow the growth of plexiform neurofibromas.

For a small subset of patients with pruritus due to cutaneous neurofibromas, diphenhydramine may provide some temporary relief. Such patients also are encouraged to avoid hot showers and baths, since hot temperatures may exacerbate itching.

Treatment with carboplatin shows efficacy in controlling the growth of visually significant optic gliomas.



Class Summary

These agents may control itching by blocking effects of endogenously released histamine.

Diphenhydramine (Aler-Dryl, Benadryl, Diphen, Altaryl)

First-generation antihistamine with anticholinergic effects that binds to H1 receptors in the CNS and the body.

Competitively blocks histamine from binding to H1 receptors. Has significant antimuscarinic activity and penetrates CNS, which causes pronounced tendency to induce sedation.

Approximately half of those treated with conventional doses experience some degree of somnolence. A small percentage of children paradoxically respond to diphenhydramine with agitation.

For symptomatic relief of pruritus caused by release of histamine in inflammatory reactions.


Antineoplastic agents

Class Summary

These agents inhibit cell growth and proliferation. The agent carboplatin has been used in the treatment of visually significant optic nerve gliomas.


Alkylating agent that has been used extensively in treatment of ovarian cancer, but with efficacy in treatment of optic nerve lesions in combination with vincristine sulfate.