OBJECTIVES: Genotropin® is a brand of somatropin (human growth hormone [GH]) licensed for the treatment of children with short stature due to: growth hormone deficiency (GHD), Prader-Willi syndrome (PWS), Turner syndrome (TS), chronic renal insufficiency (CRI) and those born small for gestational age (SGA).  Although final height (FH) is probably the most effective outcome for measuring somatropin effectiveness, there is a lack of randomised controlled trial (RCT) data reporting FH and other important outcomes, such as quality of life (QoL) which is rarely reported for children.  The objective of this systematic review (SR) of RCTs and observational studies was to investigate the efficacy and safety of Genotropin in children with these indications, and identify whether the lack of relevant RCT data in this therapy area can be supplemented with observational studies. METHODS: Predefined search terms were used to search eight electronic databases, including Medline and Embase, for published English language studies.  Additionally, bibliographies of included articles were examined for relevant studies.  RCTs or observational studies were retrieved if they included a population of children (<16 years) with GHD, PWS, TS, CRI or SGA treated with Genotropin.  The main reported outcome measures included FH and short-term growth responses, e.g. growth velocity. RESULTS: Thirty RCTs and 37 observational studies were identified.  No RCTs were identified that included data on QoL.  One RCT and 11 observational studies reported data for FH.  FH was consistently improved following treatment with Genotropin.  Seven of the observational studies were based on data sourced from the Pfizer International Growth Survey (KIGS), which showed significant gains of up to 2.3 height standard deviation scores. CONCLUSIONS: This SR reveals the paucity of long-term RCTs reporting data on FH and QoL in children, thus highlighting the consequent importance of observational studies of GH therapy, such as KIGS, which reports FH.