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General Reference

Nejm 2006;355:1345

Pathophys and Cause

Cause:Genetic, two genes: TSC1 on chromosome 9 producing humartin, and TSC2 on chromos 16 producing tuberin; autosomal dominant

Pathophys:Large hamartomas of brain, skin, et al. White spots due to decreased synthesis of melanin, but since there is a normal number of melanocytes, they are not vitiligo

Epidemiology

1/10000 population

Signs and Symptoms

Sx:Epilepsy, mental retardation in some

Si:Peri/subungual fibromas especially of toenail; angiofibromas of face (adenoma sebaceum), may look like acne without blackheads; hypopigmented macular patches ("ash leaf" macules or "white confetti") on the legs, best seen under Wood's lamp (Nejm 1998;338:1887), especially at birth, are 1st si; collagenoma of lower back skin looks like pig skin; mulberry tumors in fundus; pitted tooth enamel

Course

Variable

Complications

Seizures (75%); renal failure due to angioleiomyoma and angiomyolipomas on ultrasound and CT (Nejm 1998;338:1886); honeycomb lung; rhabdomyosarcoma of heart (Nejm 1967;276:957); hamartomas throughout body

Lab and Xray

Xray:MRI/CT of head shows calcified tumors or gliomas, subependymal tubers (Nejm 1998;338:1886)

Treatment

Rx:Monitor for cmplc