growth%20hormone%20deficiency%20(pediatric)
GROWTH HORMONE DEFICIENCY (PEDIATRIC)
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).

Growth%20hormone%20deficiency%20(pediatric) Treatment

Principles of Therapy

Growth Hormone (GH) Replacement Therapy

  • GH replacement therapy is best accomplished under the direct supervision of a pediatric endocrinologist
  • Goals of pharmacotherapy
    • To restore normal GH levels and to reduce morbidity
    • To enable short children to achieve normal height with early improvement of the psychosocial problems related to short stature

Pharmacotherapy

Somatropin, Growth Hormone (GH), or Recombinant Human Growth Hormone (rhGH)

  • Synthetic polypeptide human GH of recombinant DNA (rDNA) origin
  • Indicated for children with growth hormone deficiency (GHD), Turner syndrome, Prader-Willi syndrome, Noonan syndrome, short children small for gestational age, children with short stature homeobox-containing gene (SHOX) deficiency, and children with impaired growth due to chronic kidney disease
  • May also be used for children with idiopathic short stature (ISS) shorter than -2.25 SD score whose epiphyses are not closed, and with expected adult height below the normal range [160 cm (<63 in) for males, 150 cm (<59 in) for females]
  • Treatment should be started as soon as diagnosis of GHD is confirmed
  • GH trial therapy also recommended for children with otherwise unexplained short stature who pass GH stimulation tests, but who meet most of the following criteria:
    • Height >2.25 SD below the mean for age or >1.5 SD below the midparental height percentile
    • Growth velocity <25th percentile for bone age
    • Bone age >2 SD below the mean for age
    • Low serum IGF-1 and IGFBP-3
    • Other clinical features suggestive of GHD
  • Effects: Induce normal statural growth and correction of hypoglycemia
  • Target for treatment:
    • Satisfactory response is defined as an increase of height velocity of at least 2-2.5 cm/year above pre-treatment velocity
    • Achieve normal adult height
    • Attainment of bone age of 14 years (girls) and 16 years (boys)
  • Treatment should be started as soon as diagnosis of GHD is confirmed
  • Weight-based or body surface area (BSA)-based dosing is recommended  
  • Further studies are needed to prove the relationship between GH therapy during childhood and increased incidence of stroke in adolescents; preventive measures against stroke is advised for those with known risk factors

Gonadotropin-Releasing Hormone (GnRH) Agonist

  • Studies found GnRH agonist to be effective when given concurrently with recombinant GH
  • May delay epiphyseal fusion and prolong growth during puberty
  • Increases height and velocity in some GHD pediatric patients

Recombinant IGF-1/Mecasermin

  • Alternative treatment for patients with GH receptor abnormalities, STAT5b gene defects, and those with severe GHD unresponsive to therapy due to development of antibodies against GH
  • Further studies are needed to prove its efficacy in GHD
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