Type 1 Neurofibromatosis Medication

  • Author: Beth A Pletcher, MD; Chief Editor: Amy Kao, MD   more...
 
Updated: Feb 6, 2012
 

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.

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Antihistamines

Class Summary

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

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Diphenhydramine (Benadryl, Benylin, Diphen, AllerMax)

 

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.

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Alpha-adrenergic blocking agents

Class Summary

These agents cause vasodilation of veins and arterioles and decrease total peripheral resistance and blood pressure.

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Prazosin (Minipress)

 

Postsynaptic alpha1-antagonist, decrease blood pressure with minimal risk of reflex tachycardia.

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Doxazosin (Cardura)

 

Quinazoline compound selective alpha1-adrenergic antagonist. Inhibits postsynaptic alpha-adrenergic receptors, resulting in vasodilation of veins and arterioles and decrease in total peripheral resistance and blood pressure.

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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.

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Erlotinib (Tarceva)

 

Pharmacologically classified as a Human Epidermal Growth Factor Receptor Type 1/Epidermal Growth Factor Receptor (HER1/EGFR) tyrosine kinase inhibitor. EGFR is expressed on the cell surface of normal cells and cancer cells.

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Sorafenib (Nexavar)

 

First oral multikinase inhibitor that targets serine/threonine and tyrosine receptor kinases in both the tumor cell and the tumor vasculature. Targets kinases involved in tumor cell proliferation and angiogenesis, thereby decreasing tumor cell proliferation. These kinases included RAF kinase, VEGFR-2, VEGFR-3, PDGFR-beta, KIT, and FLT-3. Indicated for renal cell carcinoma.

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Carboplatin (Paraplatin)

 

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.

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Contributor Information and Disclosures
Author

Beth A Pletcher, MD  Associate Professor, Co-Director of The Neurofibromatosis Center of New Jersey, Department of Pediatrics, University of Medicine and Dentistry of New Jersey

Beth A Pletcher, MD is a member of the following medical societies: American Academy of Pediatrics, American College of Medical Genetics, American Medical Association, and American Society of Human Genetics

Disclosure: Nothing to disclose.

Specialty Editor Board

Ann M Neumeyer, MD  Medical Director, Lurie Family Autism Center/LADDERS; Assistant Professor of Neurology, Harvard Medical School; Child Neurologist, Massachusetts General Hospital

Ann M Neumeyer, MD is a member of the following medical societies: American Academy of Neurology, Child Neurology Society, and Massachusetts Medical Society

Disclosure: Nothing to disclose.

Francisco Talavera, PharmD, PhD  Adjunct Assistant Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Medscape Salary Employment

Kenneth J Mack, MD, PhD  Senior Associate Consultant, Department of Child and Adolescent Neurology, Mayo Clinic

Kenneth J Mack, MD, PhD is a member of the following medical societies: American Academy of Neurology, Child Neurology Society, Phi Beta Kappa, and Society for Neuroscience

Disclosure: Nothing to disclose.

Chief Editor

Amy Kao, MD  Attending Neurologist, Children's National Medical Center

Amy Kao, MD is a member of the following medical societies: American Academy of Neurology, American Academy of Pediatrics, American Epilepsy Society, and Child Neurology Society

Disclosure: Nothing to disclose.

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Café-au-lait spots in a 4-year-old boy.
Axillary freckles.
Inguinal freckles.
Multiple neurofibromas in a 28-year-old man.
Plexiform neurofibroma of the right thigh.
Lisch nodules.
Radial and ulnar bowing and obliteration of the intramedullary spaces.
Unidentified bright object (UBO) within the brain parenchyma.
Left optic nerve glioma with thickening of the nerve and proptosis.
Below-the-knee amputation for tibial pseudarthrosis.
The young woman pictured here has a plexiform neurofibroma of the eyelid.
 
 
 
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