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

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

Somatropin, Growth Hormone, 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 inches) for males, 150 cm (<59 inches) 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 are 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 GH-deficient 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