Pediatric Growth Hormone Deficiency Guidelines

Updated: Jun 21, 2018
  • Author: Vaneeta Bamba, MD; Chief Editor: Robert P Hoffman, MD  more...
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Guidelines

Guidelines Summary

Guidelines on growth disorders and their treatment by the Drug and Therapeutics Committee and Ethics Committee of the Pediatric Endocrine Society  [21]

  • Use growth hormone (GH) to normalize adult height (AH) and avoid extreme shortness in children and adolescents with growth hormone deficiency (GHD).
  • Suggest against routine cardiac testing, dual x-ray absorptiometry (DXA) scanning, and measurement of lipid profiles in children and adolescents treated with GH.
  • Establish a diagnosis of GHD without GH provocative testing in patients possessing all of the following 3 conditions: auxological criteria, hypothalamic-pituitary defect (such as major congenital malformation [ectopic posterior pituitary and pituitary hypoplasia with abnormal stalk], tumor, or irradiation), and deficiency of at least one additional pituitary hormone.
  • GHD due to congenital hypopituitarism should be diagnosed without formal GH provocative testing in a newborn with hypoglycemia who does not attain a serum GH concentration above 5 µg/L and has deficiency of at least one additional pituitary hormone and/or the classical imaging triad (ectopic posterior pituitary and pituitary hypoplasia with abnormal stalk).
  • Recommend against reliance on GH provocative test results as the sole diagnostic criterion of GHD.
  • Suggest sex steroid priming prior to provocative GH testing in prepubertal boys older than 11 yr and in prepubertal girls older than 10 yr with AH prognosis within -2 SD of the reference population mean in order to prevent unnecessary GH treatment of children with constitutional delay of growth and puberty.
  • Recommend against the use of spontaneous GH secretion in the diagnosis of GHD in a clinical setting.
  • Recommend an initial GH dose of 0.16-0.24 mg/kg/wk (22-35 µg/kg/day) with individualization of subsequent dosing.
  • Suggest measurement of serum insulin-like growth factor-I (IGF-I) levels as a tool to monitor adherence and IGF-I production in response to GH dose changes. Suggest that the GH dose be lowered if serum IGF-I levels rise above the laboratory-defined normal range for the age of pubertal stage of the patient.
  • During puberty, recommend against the routine increase in GH dose to 0.7 mg/kg/wk in every child with GHD.
  • Recommend that GH treatment at pediatric doses not continue beyond attainment of a growth velocity below 2-2.5 cm/yr. The decision to discontinue pediatric dosing prior to attainment of this growth velocity should be individualized.
  • Recommend that prospective recipients of GH treatment receive anticipatory guidance regarding the potential adverse effects of intracranial hypertension, slipped capital femoral epiphysis (SCFE), and scoliosis progression.
  • Recommend monitoring of GH recipients for potential development of intracranial hypertension, SCFE, and scoliosis progression by soliciting pertinent history and performing a physical examination at every follow-up clinic visit; further testing should be pursued if indicated.
  • Recommend re-assessment of both the adrenal and thyroid axes after initiation of GH therapy in patients whose cause of GHD is associated with possible multiple pituitary hormone deficiencies (MPHD).
  • For GH initiation after completion of tumor therapy with no evidence of ongoing tumor, a standard waiting period of 12 mo to establish “successful therapy” of the primary lesion is reasonable, but can also be altered depending on individual patient circumstances.
  • Recommend that patients with multiple (≥3) pituitary hormone deficiencies regardless of etiology, or GHD with a documented causal genetic mutation or specific pituitary/hypothalamic structural defect except ectopic posterior pituitary, be diagnosed with persistent GHD.
  • Recommend re-evaluation of the somatotropic axis for persistent GHD in persons with GHD and deficiency of only one additional pituitary hormone, idiopathic isolated GHD (IGHD), IGHD with or without small pituitary/ectopic posterior pituitary, and after irradiation.
  • Suggest that measurement of the serum IGF-I concentration be the initial test of the somatotropic axis if re-evaluation of the somatotropic axis is clinically indicated.
  • Recommend GH provocative testing to evaluate the function of the somatotropic axis in the transition period if indicated by a low IGF-I level.
  • Suggest that GH treatment be offered to individuals with persistent GHD in the transition period. There is evidence of benefit; however, the specifics of the patient population that benefits, the optimal time to re-initiate treatment, and the optimal dose are not clear.
  • Because there is overlap in response between dosing groups, suggest initiating GH at a dose of 0.24 mg/kg/wk, with some patients requiring up to 0.47 mg/kg/wk.
  • Recommend the use of IGF-I therapy to increase height in patients with severe primary IGF-I deficiency (PIGFD).
  • Recommend a trial of GH therapy before initiating IGF-I for patients with unexplained IGF-I deficiency. Patients with hormone signaling defects known to be unresponsive to GH treatment can start directly on IGF-I replacement; these include patients with very low or undetectable levels of GH-binding protein (GHBP) and/or proven GH receptor (GHR) gene mutations known to be associated with Laron syndrome/GH insensitivity syndrome (GHIS), GH-neutralizing antibodies, STAT5b gene mutations, and IGF1 gene deletion or mutation.
  • Suggest an IGF-I dose of 80-120 µg/kg BID. Similar short-term outcomes were seen with 80 and 120 µg, but published studies had limitations, and there is no strong evidence supporting superiority of one dose over the other.