Growth hormone for short children without a hormone deficiency: Issues and practices

October 1, 2005

Five new indications for growth hormone in children who are not GH-deficient have gained FDA approval. What are the pros and cons?

Recombinant growth hormone (GH) was first made available in 1985 and was initially used exclusively to treat children with short stature caused by growth hormone deficiency. In the ensuing years, however, the use of GH as a pharmacologic agent to improve linear growth in short children without GH deficiency has been explored, leading to approval by the Food and Drug Administration of a number of indications for GH use in non-GH-deficient children. Children with chronic renal insufficiency, Turner syndrome, or Prader-Willi syndrome, children who are born small for gestational age, and children with idiopathic short stature (Table 1) are all now eligible for recombinant GH therapy.

An overview of GH therapy

Route of administration. GH is given daily by subcutaneous injection, and parents are trained by health-care providers to administer injections.1 Previously, GH was given three times a week, but it is now recommended that it be given six or seven times a week because studies have shown that outcomes are improved with more frequent dosing.2

Duration of treatment. On average, the mean age of initiation of GH treatment is around 10 years,3 and because GH is usually given until children reach final adult height, treatment can last five years or longer.4,5

Contraindications. GH is contraindicated for children who have active malignancies or closed epiphyses.

Other serious adverse events, including slipped capital femoral epiphysis (SCFE) and benign intracranial hypertension (IH), have been reported but are rare. Any child receiving GH who complains of knee or hip pain or a limp should be evaluated for SCFE. Any child on GH who complains of visual changes, headache, nausea, or vomiting should be assessed for papilledema and IH, especially within the first few months after initiating treatment.4,5 Fortunately, IH is reversible, and GH can usually be restarted at lower doses.7