Clinical effectiveness and cost-effectiveness of growth hormone in children: a systematic review and economic evaluation

Results Number and quality of studiesRCTs comparing GH with placebo or no treatment were included, and because final height data were rarely available in the context of RCTs, lower levels of evidence were included for final height only, using the highest level of evidence available within each condition.A total of 34 publications reporting 32 studies were included in the assessment of clinical effectiveness.Short-term growth and final height outcomes were evaluated along with some body composition and psychological outcomes.The Jadad quality scores of the trials ranged from 1/5 to 4/5.No existing economic evaluations were found, nor were there any studies reporting appropriate measures of quality of life. Summary of benefitsAlthough the quality of evidence proved variable, the studies suggest that GH treatment can increase short-term growth and improve final height.The reported effects of GH on short-term growth should be considered more reliable because the evidence is of higher quality.The effects of GH on final height should be considered with much greater caution because the quality of the studies is generally much poorer.Results suggest that the effects of GH on shortterm growth velocity (at 1 year) can range from no improvement to approximately 1 standard deviation above the normal growth velocity for children of the same age.Final height gains for treated children over untreated children appear to range from approximately 2 to 11 cm (GHD, 8-11 cm; TS, 5 cm; CRF, 3-9 cm; PWS, 10-11 cm; ISS, 2-7 cm). CostsTreatment with GH is expensive.The lifetime incremental cost of treating one child with GH (as opposed to simply monitoring growth) ranges from £43,100-53,400 (for GHD) to £55,500-83,000 (for PWS).These costs, when applied to children aged 8-15 years with the analysed indications in England and Wales, result in total discounted costs of £904 million for complete treatment.The costs for treating children only in the four licensed conditions would be approximately £180 million. Cost per centimetre gainedThe available data suggest that, under base case conditions, the incremental cost per centimetre gained in final height is approximately £6000 for GHD, £16,000-17,400 for TS, £7400-24,100 for CRF, £13,500-27,200 for ISS and possibly in the region of £7030 for PWS (estimated using year 2000 prices).