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tuberous_sclerosis [2025/02/23 23:16] Scott Larsontuberous_sclerosis [2025/06/14 03:39] (current) – [Resources] Scott Larson
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-====== Tuberous Sclerosis ======+====== Tuberous Sclerosis Complex ======
  
 ====Main Features==== ====Main Features====
-  *  +  * Benign tumors of the brain, kidney, lung, heart and eyes.  
 +  * One of the [[phakomatoses|phakomatoses]]
  
 ====Eye Findings==== ====Eye Findings====
-  * +  * Retinal astrocytic hamartomas 34-75% of patients with TSC.  
 +    * flat, translucent lesions, multinodular "mulberry" lesions, and transitional types.  
 +    * usually non-progressive but can occasionally show subretinal fluid and progression.  
 +    * Typically located along the arcades, adjacent to the optic nerve, or in the retinal periphery. 
 +    * {{::retinal_hamartoma.jpg|}} 
 +  * Retinal achromic patches 34% 
 +    * hypopigmented areas similar to the hypomelanotic macules seen on the skin 
 +    * Usually non-progressive and do not significantly affect vision 
 +  * Refractive errors (myopia, hyperopia, and astigmatism),  
 +  * Strabismus 
 +  * Angiofibromas of the eyelids- rare 
 +  * Coloboma- rare 
 ====Other Findings==== ====Other Findings====
-  * +  * Brain (leading cause of morbidity) 
 +    *subependymal nodules 
 +    *cortical tubers 
 +    *subependymal giant cell astrocytomas 
 +    *Seizures 
 +    *neuropsychiatric disorders/ developmental delay 
 +  * Cardiac  
 +    * rhabdomyomas 
 +    * arrhythmias 
 +  * Renal (leading cause of mortality) 
 +    * benign renal angiomyolipomas 
 +    * epithelial cysts 
 +    * oncocytoma 
 +  * Lungs 
 +    * lymphangioleiomyomatosis  
 +    * multifocal micro odular pheumonocyte hyperplasia 
 +  * Dermatologic 
 +    * Angiofibromatosis 
 +    * Shagreen patches 
 ====Etiology==== ====Etiology====
-  *  +  * Autosomal Dominant with near complete penetrance but variable expressivity due to Knudson's two-hit hypothesis 
-====Reference==== +  * Mutations result in unregulated cell growth leading to hamartomas 
-  * [[link|Title]]+  * Mutations in TSC1 (25%) 
 +    * Encodes for gene hamartin- a tumor supressor gene 
 +    * Chromosome 9q34   
 +  * Mutations in TSC2 (75%) 
 +    * Encodes for gene tuberin- a tumor supressor gene 
 +  * Both genes inhibit the mammalian large of rapamycin (mTOR) pathway  
 + 
 +====Resources==== 
 +  * [[https://www.ncbi.nlm.nih.gov/books/NBK1220/|Tuberous Sclerois Complex in Gene Reviews]] 
 +  * [[https://1drv.ms/b/c/31d83ae8e55e0542/EVD_lW3jlI1Hl-7598XM2iMBGFZ1InOEprGzgt5JpvlOBw?e=uMxmAq | Hodgson N et al. Ophthalmic manifestations of tuberous sclerosis: a review. Clinical and Experimental Ophthalmology 2017;45:81-86]] 
 +  * [[https://1drv.ms/b/c/31d83ae8e55e0542/EbK3lI7wIf1Fs2bMrAdeMzoBIWMDSLAcNTl04LrjuawFSA?e=2iRKDA|Larson, SA. Lecture: Peditric Primary Intraocular Tumors ]]
  
-{{tag>syndrome}}+{{tag>syndrome phakomatosis}}