pediatrics
Growth Hormone Deficiency in Children
Treatment of Growth Hormone Deficiency
Recombinant GH supplements Sometimes other pituitary hormone replacement Recombinant GH is indicated for all children with short stature who havedocumented growth hormone deficiency. (See also the Drug and Therapeutics, and Ethics Committees of the Pediatric Endocrine Society's2016 guidelines for growth hormone and insulin-like growth factor-I treatment in children and adolescents.) Documented growth hormone deficiency is based on auxologic, biochemical, and sometimes radiologic findings. Dosing of recombinant GH is usually from 0.03 to 0.05 mg/kg subcutaneously once a day. With therapy, height velocity often increases to 10 to 12 cm/year in the first year and, although it increases more slowly thereafter, remains above pretreatment rates. Therapy is continued until an acceptable height is reached or growth rate falls below 2.5 cm/year. A preparation of GH (lonapegsomatropin) is available for patients with GH deficiency who are ≥ 1 year of age who weigh at least 11.5 kg. A typical starting dosage is 0.24 mg/kg subcutaneously once/week. Adverse effects of GH therapy are few but include Idiopathic Intracranial Hypertension (pseudotumor cerebri), It is controversial whethershort children without evidence of an endocrine, metabolic, or other disease that explains the short statureshould be treated with GH. These children are considered to have idiopathic short stature (ISS). ISS is defined as height 2 standard deviations (SD) below the mean for age, normal height velocity (near or at lower limit of normal), no biochemical evidence of a growth-restricting condition, and normal GH stimulation testing that excludes classic GH deficiency. Recombinant GH can be used to treat children with ISS who have a height 2.25 SD below the mean for age and a predicted adult height below the normal range (ie, < 150 cm for females and < 160 cm for males). Guidelines recommend against the routine use of GH for every child with ISS, and the decision to treat should be made on a case-by-case basis. Treatment responses are highly variable. With 5 years of treatment, some children may have a mean increase of about 5 cm in adult height, whereas other children may have no increase in adult height. A greater response to GH treatment may be expected in children with ISS based on height response in first year of treatment, age at the start of treatment (better response if treatment is started before age 9 in girls and before age 10 in boys), and change in IGF-1 level from baseline. For children who are treated, many experts recommend a trial of GH therapy for 6 to 12 months, continuing GH only if there is a doubling of or an increase of 3 cm/year over the pretreatment height velocity. Others object to this approach because it is expensive, is experimental, may lead to adverse effects, labels otherwise healthy children as abnormal, and raises ethical and psychosocial concerns that feed into the bias of “heightism.” accompany growth hormone deficiency, additional hormone replacement is required. Cortisol ( see Treatment ) and thyroid hormone ( see GH therapy in children with short stature due to therapeutic radiation of the pituitary gland for cancer carries a theoretic risk of causing cancer recurrence. However, studies have not shown a greater-than-expected incidence of new cancers or a greater recurrence rate. GH replacement can probably be safely instituted at least 1 year after the successful completion of anticancer therapy. 