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Updated: Dec 24 2021

21-Hydroxylase Deficiency

Images
https://upload.medbullets.com/topic/120573/images/ambiguous genitalia.jpg
https://upload.medbullets.com/topic/120573/images/virilized genitalia.jpg
https://upload.medbullets.com/topic/120573/images/steroidogenesis.jpg
  • Snapshot
    • An 8-month-old female presents with ambigous genitalia. Her urinary ketosteroids and 17-OH progesterone are twice the normal level. Additionally, testosterone levels are four times normal and urinary 17-hydroxycorticosteroids are decreased. Her brother had a similar disorder.
  • Introduction
    • A condition characterized by ambiguous genitalia in female
    • There is a non-classical from of this condition that may present in adolesence or adulthood with signs of hypertestosteronism
      • Most commonly females present with hirsuitism
  • Epidemiology
    • 1:15,000 children born with disorder
    • Can affect both males and females
  • Presentation
    • Symptoms
      • in females
        • abnormal mestruation cycles
        • deep voice
        • excessive hair growth
        • ambiguous genitalia (more male than female in most cases)
        • loss of vigor
        • loss of appetite
      • in males
        • no phenotypic problems at birth
        • may enter puberty early, sometimes at 2-3 years of age
          • deep voice
          • early, excessive hair growth
          • early development of male secondary sexual characteristics including
            • enlarged penis
            • small testes
            • muscular development
    • Physical exam
      • in females
        • usually normal reproductive organs (i.e. uterus, ovaries, fallopian tubes)
        • hirsuit (male distribution)
        • signs of hypoglycemia
        • renal dysfunction secondary to hypocortisolism and hypoaldosteronism
          • salt loss
          • volume depletion
      • in males, as above
      • in both
        • above average growth as child
        • final height usually smaller than normal adult
      • adrenal crisis
        • may develop in some newborns due to salt loss and can result in
          • dehydration
          • electrolyte changes
          • cardiac arrhythmia
          • vomiting
  • IMAGING
    • X-ray
      • may demonstrate "older bone age" than true age
  • STUDIES
    • Labs
      • decreased cortisol and aldosterone
      • increased 17-OH progesterone
      • increased serum DHEA sulfate
      • increased 17-ketosteroids
    • Urinary 17-hydroxycorticosteroids
      • normal or decreased
      • in contrast to 11-Hydroxylase Deficiency
    • Chemistry
      • electrolyte abnormalities may be evident at infancy
    • EKG
      • may show cardiac arrhythimia(s) secondary to renal dysfunction
    • Genetic testing
      • may diagnose or confirm suspected diagnosis
      • may determine genotypic gender of child to guide treatment
  • Differential
    • 11-Hydroxylase deficiency, 17-hydroxylase deficiency
  • Treatment
    • Prevention
      • genetic counseling may help parents with family history of disorder
      • there is no preventive measure available at this time
    • Medical management
      • control cortisol levels
        • goal is to achieve normal cortisol levels
        • dexamethasone, fludrocortisone, or hydrocortisone may be indicated
      • determine gender
        • checking karyotype can accomplish this task
    • Surgical management
      • gender assignment surgery
        • based on karyotypic findings
        • females usually undergo surgery 1-3 months of age to remove male anatomy
  • Complications
    • Shortened adult height, ambiguous genitalia, hypertension, hypoglycemia, smaller vaginal introitus, death via adrenal crisis if untreated, steroid side effects, patients medicated for life, male testicular tumors
  • Prognosis
    • Good to very good in most individuals
    • If untreated, adrenal crisis can lead to death within 1-6 weeks
    • Males usually have normal fertility; females may be abnormal fertility
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