Systematic review of the clinical effectiveness of Genotropin (somatropin) in children with short stature.

Genotropin (somatropin) is licensed for the treatment of children with growth hormone deficiency, Prader-Willi syndrome, Turner syndrome, chronic renal insufficiency and in children born small for gestational age. This systematic review (SR) evaluated the clinical efficacy and effectiveness of Genotropin in these conditions to inform a NICE Technology Appraisal of growth hormone for the treatment of growth failure in children. Search terms were used to search seven databases, including Medline and Embase, for English language studies. Randomised controlled trials (RCTs) or observational studies investigating Genotropin in children were included. Out of 30 RCTs identified, one reported final height data. Eleven observational studies reported final height and seven were based on the Pfizer International Growth Survey (KIGS). This SR highlights the lack of long-term RCTs reporting final height data and other important qualitative outcomes, such as quality of life. Observational data, such as those from KIGS, remain vital for informing therapy.