Growth hormone deficiency is a congenital or an acquired growth hormone axis disruption in the higher brain, hypothalamus or pituitary which results in short stature.
It may occur at any age.
Short stature is often the only feature present in patients with growth hormone deficiency and other causes of subnormal growth rate.
Etiology may be congenital conditions (eg defective pituitary development that leads to pituitary aplasia, empty sella, encephalocele, midline defects) or acquired conditions (eg tumors of the hypothalamic-pituitary region, cranial irradiation, infiltrative disease).


Prescribed End Points

  • IGF-I in normal range for age & sex
    • Increase dose if IGF-I is low & decrease dose if IGF-I is above normal range
  • Improvement in blood lipid profile, body composition (change in lipolysis & increase in bone density) & waist-hip ratio
  • Increased muscle strength & exercise performance
  • Reduction in CV risk factors

Patient Monitoring

  • Close follow-up care w/ an endocrinologist is recommended to monitor the child’s growth & to adjust the dose of growth hormone (GH) therapy
  • Initial follow-up should be every month; thereafter, visits may be less frequent but should be at least 2x/year
  • Children on GH therapy should be evaluated every 3-6 months, w/ increases in height & height velocity as the most important indicators of response
  • Monitor thyroid function every 6 months
  • Bone age, HbA1c levels, & adrenal function should also be assessed regularly
  • Monitor patients for hyperglycemia because GH may reduce insulin sensitivity; patients w/ diabetes mellitus (DM) may need to adjust their insulin during treatment
  • Serum IGF-1 & IGFBP-3 levels should be obtained annually
  • Funduscopic examination is recommended at the start of therapy & periodically during the course of treatment
  • GH treatment is meant to be a replacement therapy & can only be expected to make short children grow at a normal growth rate
  • Most children treated w/ GH replacement reach a normal adult height
    • Child’s growth usually increases most during the 1st years, w/ an average increase of 8-10 cm/year
    • Growth rate slows down over the next several years
  • Re-evaluation should be done once patient completed
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