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Neurofibromatosis Type 1 Medication

  • Author: David T Hsieh, MD, FAAP; Chief Editor: Amy Kao, MD  more...
 
Updated: Jul 27, 2016
 

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.

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.

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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.

Carboplatin

 

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

David T Hsieh, MD, FAAP Assistant Professor of Pediatrics, Assistant Professor of Neurology, Uniformed Services University of the Health Sciences, F Edward Hebert School of Medicine; Adjunct Assistant Professor of Pediatrics, Adjunct Assistant Professor of Neurology, University of Texas Health Science Center at San Antonio School of Medicine

David T Hsieh, MD, FAAP is a member of the following medical societies: American Academy of Neurology, American Academy of Pediatrics, American Epilepsy Society, Child Neurology Society

Disclosure: Nothing to disclose.

Coauthor(s)

Luis O Rohena, MD Chief, Medical Genetics, San Antonio Military Medical Center; Assistant Professor of Pediatrics, Uniformed Services University of the Health Sciences, F Edward Hebert School of Medicine; Assistant Professor of Pediatrics, University of Texas Health Science Center at San Antonio

Luis O Rohena, MD is a member of the following medical societies: American Academy of Pediatrics, American Chemical Society, American College of Medical Genetics and Genomics, American Society of Human Genetics

Disclosure: Nothing to disclose.

Specialty Editor Board

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

Disclosure: Received salary from Medscape for employment. for: Medscape.

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, 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 Epilepsy Society, Child Neurology Society

Disclosure: Have stock from Cellectar Biosciences; have stock from Varian medical systems; have stock from Express Scripts.

Additional Contributors

Ann M Neumeyer, MD Medical Director, Lurie Center for Autism; 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, Massachusetts Medical Society

Disclosure: Nothing to disclose.

Acknowledgements

The view(s) expressed herein are those of the author(s) and do not reflect the official policy or position of Brooke Army Medical Center, the U.S. Army Medical Department, the U.S. Army Office of the Surgeon General, the Department of the Army, the Department of the Air Force, Department of Defense or the U.S. Government.

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.

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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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