Therapy In Practice

Pediatric Drugs

, Volume 10, Issue 5, pp 299-313

First online:

Current Management of Tuberous Sclerosis Complex

  • Darcy A. KruegerAffiliated withTuberous Sclerosis Clinic, University of Cincinnati College of Medicine, Cincinnati Children’s Hospital Medical Center
  • , David N. FranzAffiliated withTuberous Sclerosis Clinic, University of Cincinnati College of Medicine, Cincinnati Children’s Hospital Medical Center Email author 

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Abstract

Tuberous sclerosis complex (TSC) is an important cause of epilepsy, autism, and renal and pulmonary disease in children and adults. The clinical course of TSC and the prognosis and appropriate therapy for TSC patients are often different than that for individuals with epilepsy, renal tumors, or interstitial lung disease from other causes. This article reviews the current therapeutic recommendations for medical and surgical management of neurologic, renal, and pulmonary manifestations of TSC. In addition, recent clinical trials using inhibitors of the mammalian target of rapamycin (mTOR) have demonstrated regression of astrocytomas, angiofibromas, and angiomyoliomas, as well as improved pulmonary function in persons with TSC.