A drug can make children with dwarfism taller

What the research found

Clinical guidelines cover growth hormone treatment for children with growth hormone deficiency, idiopathic short stature (short with no clear cause) and one type of insulin-like growth factor deficiency. A 2013 New England Journal of Medicine review reported that growth hormone can add 3 to 7 cm to adult height in children with idiopathic short stature, with wide variation between children.

A randomised trial from 2000 tested growth hormone in children with achondroplasia, a common cause of severe dwarfism. Growth rate and height scores rose, and the effect was larger at the higher dose. No adverse effects were seen in either dose group. The authors concluded that growth hormone is a useful treatment for the severe growth problems of achondroplasia. The trial reported growth rate and height scores, not final adult height.

Not every type of dwarfism responds. In a systematic review of 42 people with SOFT syndrome, a rare form of dwarfism, growth hormone treatment was stopped in 7 of 9 patients because they grew little or not at all.

What it doesn’t mean

• This does not mean a drug will work for every child with dwarfism. The cause of the short stature matters, and some children gain little or nothing.
• Growth hormone is a prescription treatment that needs specialist assessment. Gains vary between children, and the treatment is expensive, so the likely benefit should be weighed against the risks and costs for each child.
• If you are worried about your child's growth, see your GP or child health nurse. They can arrange tests and a referral to a paediatric endocrinologist if needed.