6 Recommendations for further research


The following trials are currently ongoing:

  • Study NCT00190658 aims to compare the mean first year growth velocity of prepubertal children with SHOX deficiency treated with somatropin with the growth velocity of a control group of untreated prepubertal children with SHOX deficiency. Estimated end date: December 2010.

  • Study NCT00625872 focuses on the effect of a 1‑year somatropin treatment (35 microgram/kg per day or 67 microgram/kg per day) in short children born small for gestational age on neuromuscular function and cognitive performance. End date not reported.

  • There is a controlled cohort study examining health‑related quality of life in family members of children prescribed growth hormone treatment for idiopathic growth hormone deficiency, acquired growth hormone deficiency and Turner syndrome. In September 2009, one of the investigators informed NICE that results were not expected until the end of 2010.


A standardised quality‑of‑life assessment measuring quality of life in children and in adults is needed for use in future RCTs and studies designed to measure quality of life.


Good quality research is needed on the long‑term effects of somatropin treatment during childhood on body composition, psychological health, diabetes, cardiovascular disease and bone health, and life expectancy, particularly for people with Prader–Willi syndrome.


Good quality research is needed on somatropin treatment in short children born small for gestational age using dosages of somatropin matching the licensing criteria.

  • National Institute for Health and Care Excellence (NICE)