In children with idiopathic short stature, what advantage does administering recombinant growth hormone have over observation in final adult height?: Part B: Clinical commentary.

It is not uncommon for the families of children with idiopathic short stature (ISS) to inquire about recombinant human growth hormone (rhGH) treatment. rhGH became available in 1985 shortly after cadaveric human growth hormone (hGH) was withdrawn from distribution secondary to associated cases of Creutzfeld-Jacob disease. Before the availability of rhGH, hGH therapy was primarily limited to patients with growth hormone deficiency. The unlimited supply of rhGH opened up new possibilities for increased doses and indications for treatment. ISS is not an approved indication for rhGH in Canada, but it has recently been approved as an indication by the United States Food and Drug Administration. In Canada, rhGH use is approved for growth hormone deficiency in children and adults, Turner syndrome, chronic renal insufficiency in children before transplantation, and human immunodeficiency virus-associated wasting syndrome in adults. Additional approved indications in the United States include Prader-Willi syndrome, and children born small for gestational age who remain small at two years of age (1,2). rhGH is given by subcutaneous injection. Its side effects are rare but include local reactions at the injection site, transient peripheral edema, pseudotumor cerebri, slipped capital femoral epiphysis, scoliosis and impaired glucose tolerance (1,3). The annual costs for a 30 kg child on a conservative dose is approximately CDN$12,000 to CDN$16,000. In Canada, various combinations of provincial health plans, private insurance and compassionate coverage by industry cover the costs of rhGH. The assessment of rhGH trials is difficult because of the limited number of randomized control trials, and nonstandardized inclusion criteria, dosing and outcome measures. As reviewed by Chan and Klassen (pages 569–570), rhGH therapy may provide a small increase in final adult height in children with ISS. However, this increased height does not necessarily result in an improved quality of life for these children (4). The use of rhGH in ISS is a contentious issue. rhGH therapy brings with it the risk of practicing ‘cosmetic endocrinology’ (5). Does the possibility of a small increase in height warrant therapy in a healthy normal child? How many dollars or daily injections is an additional few centimetres worth? Does the benefit of an additional few centimetres differ depending on the absolute height? Should only those with private health insurance have access to therapy or should costs be covered through provincial health plans? As a society, we must weigh the costs and benefits of the use of rhGH in ISS and other conditions.

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