Use of growth hormone (GH) in non-GH deficient short stature

作者：Menon P S N^*

来源：Indian Journal of Pediatrics, 2008, 75(1): S14-S19.

• achondroplasia
• dyschondrosteosis
• growth hormone
• hypochondroplasia
• idiopathic short stature
• insulin-like growth factor-1
• Prader-Willi syndrome
• skeletal dysplasia
• X-linked hypophosphatemic rickets

In the last two decades growth hormone (GH) therapy has expanded to include many children with non-GH deficient short stature such as idiopathic short stature (ISS), skeletal dysplasia, genetic syndromes and other chronic diseases associated with short stature. ISS now appears to be the most common indication for GH treatment. It is difficult to differentiate GHD from ISS with conventional GH testing alone. A number of abnormalities of the GH receptor have been identified in children with ISS. GH in a supraphysiological dosage generally increases height velocity in children with ISS and increases the adult height upto 7 cm. However, the height gain for an individual child cannot be predicted. Though GH is well tolerated without significant adverse effects, the risks of unwanted long-term sequelae of elevated serum GH and IGF-1 have not been evaluated. The psychosocial effects of short stature and the emotional benefits derived from GH therapy also need to be carefully evaluated. The results in skeletal dysplasia have been less rewarding. GH therapy does not benefit patients with achondroplasia much, but a subgroup of patients with hypochondroplasia may benefit significantly. Other uncommon forms of skeletal dysplasias have not benefited with GH treatment. The compromised final height, motor performance, muscle mass, the body composition and metabolic profile improve with GH therapy in Prader-Willi-Syndrome (PWS). However, these children need close monitoring for respiratory difficulties and apnea, disturbance of carbohydrate metabolism and scoliosis. In view of its safety, the use of GH is likely to be extended to many disorders where short stature may be an associated secondary problem. In future the use of growth hormone for disorders with non-GHD short stature is likely to increase further.

• 出版日期2008-1