Tuberous sclerosis

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Treatments and drugs

By Mayo Clinic staff

There's no cure for tuberous sclerosis, but treatment can help manage specific signs and symptoms. For example:

  • Medication. Anti-epileptic drugs may be prescribed to control seizures, and other medications may be prescribed to help manage behavior problems. A medication called everolimus (Afinitor, Zortress) is used to treat certain types of brain growths that can't be surgically removed in people with tuberous sclerosis. Another medication that may prove helpful for brain growths and other problems caused by tuberous sclerosis is an immune-suppressing drug called sirolimus (Rapamune). The topical form of this drug may help treat the acne-like skin lesions that can occur in tuberous sclerosis. However, sirolimus is still in clinical trials and considered experimental as a treatment for tuberous sclerosis.
  • Educational therapy. Early intervention can help children overcome developmental delays and meet their full potential in the classroom.
  • Occupational therapy. Through occupational therapy, a person who has tuberous sclerosis can improve his or her ability to handle daily tasks.
  • Psychological therapy. Talking with a mental health therapist may help you or your child accept and adjust to living with this disorder.
  • Surgery. If a lesion affects the ability of a specific organ — such as the kidney — to function, the lesion may be surgically removed. Sometimes surgery can help control seizures caused by brain lesions that don't respond to medication. Surgical procedures such as dermabrasion or laser treatment may improve the appearance of skin lesions.

Tuberous sclerosis is a lifelong condition that requires careful monitoring and follow-up. With appropriate treatment, however, many people who have tuberous sclerosis lead full, productive lives and enjoy a normal life expectancy.

References
  1. Tuberous sclerosis fact sheet. National Institute of Neurological Disorders and Stroke. http://www.ninds.nih.gov/disorders/tuberous_sclerosis/detail_tuberous_sclerosis.htm. Accessed Oct. 4, 2011.
  2. Seibert D, et al. Recognition of tuberous sclerosis in adult women: Delayed presentation with life-threatening consequences. Annals of Internal Medicine. 2011;154:806.
  3. Tuberous sclerosis complex. http://www.uptodate.com/home/index.html. Accessed Oct. 5, 2011.
  4. My child has tuberous sclerosis: A brochure for parents. Tuberous Sclerosis Alliance. http://www.tsalliance.org/publications/52810_NTSA.pdf. Accessed Oct. 4, 2011.
  5. Tuberous sclerosis complex. Genetics Home Reference. http://ghr.nlm.nih.gov/condition/tuberous-sclerosis-complex. Accessed Oct. 4, 2011.
  6. DeKlotz CMC, et al. Dramatic improvement of facial angiofibromas in tuberous sclerosis with topical rapamycin: Optimizing a treatment protocol. Archives of Dermatology. 2011;147:1116.
  7. Patterson MC (expert opinion). Mayo Clinic, Rochester, Minn. Oct. 8, 2011.
DS01032 Nov. 1, 2011

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