lecture 4 tyrosinemia Flashcards


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1

what is tyrosinemia ?

  • autosomal recessive
  • gene mutation from each parent
  • 1:4 risk the child is affected
  • organ responsible metabolism > liver
  • tyrosine > aminoacid > plant & animal

2

importance of tyrosine ?

  • essential AA
  • PRECOURCE OF thyroxine, melanin, catecholamines
  • insoluble in formulas > shaking

3

tyrosinemia type one

  • reduced activity of fumaryl aceto acetate hydrolase
  • FAH > final step of tyrosine degradation
  • > accumilation succinylacetone > damage to liver & kidneys & neurological problems

4

acute infantile form onset

  • first month of life
  • death of hepatic failure between 3-9 months

5

acute infantile form manifestations

  1. poor weight gain
  2. enlarged liver & spleen
  3. jaundice
  4. distended abdomen
  5. swollen legs
  6. cabbage like odor
  7. funconi syndrome
  8. nose bleeds
  9. liver failure > vomiting, bleeding, sepsis, hypoglycemia
  10. liver cancer

6

chronic form onset

  • before 6 months (late)
  • death by 10 years

7

chronic form manifestations

  1. poor weight gain
  2. ascites
  3. growth retardation
  4. rickets
  5. hepatomegaly
  6. cirrhosis
  7. hematoma
  8. tubulopathy
  9. neuropathy
  10. abdominal pain
  11. enlargement of liver and spleen
  12. vomiting and diarrhea

8

neurological crisis

  • metal changes
  • peripheral neuropathy
  • abdominal pain
  • respiratory failure
  • 1-7 days

9

tyrosinemia diagnosis

  • ^ succinylacetone
  • normal / ^ tyrosine
  • ^ methionine
  • ^ colics / delta-amino-levulinic acid
  • ^ alfa-feto protein

10

goals of the nutritional therapy for tyrosinemia

  1. support growth
  2. normal intellectual development
  3. adequate nutrition
  4. prevent neurological crisis
  5. prevent liver& renal failure
  6. prevent the formation of tyrosine crystals (eyes)
  7. tyrosine levels < 500 umol/l
  8. normal phenylalanine levels

11

therapy for type 1 tyrosinemia

  1. low phe and tyrosine diet
  2. low protein
  3. niti-sinone (orfadin) - 1mg/kg
  4. adequate energy > normal growth and prevent protein catabolism
  5. fat soluble vitamins supplements
  6. prevent rickets > PO4 + K + vit D supplements
  7. high methionine levels > reduce it to normal
  8. if therapy fails > live transplant

12

diet during infections for tyrosinemia patients

  • protein free
  • high energy
  • prevents > decompactions of liver > deterioration of neurological

13

tyrosinemia type 2 facts

  • enzyme defect > cytosolic tyrosine amino transferase
  • the rarest type

14

tyrosinemia type 2 characteristics

  • corneal lesions > lacrimation, photophobia, scars
  • corneal ulcers
  • hyperkeratotic lesions > digits, palms, soles
  • mild mental retardation
  • intellectual disability
  • seizures
  • intermitted ataxia

15

transient tyrosinemia definition & causes

  • 10% of newborns
  • temporarily elevated levels of tyrosine
  • not genetic
  • vit C deficincy
  • immature liver enzymes > pre term