Background
idiopathic, GHD
EARLY PUBERTY, GROWTH HORMONE DEFICIENCY, GHD
Around 500 children are diagnosed with growth hormone deficiency (GHD) every year in the UK, of whom 75% have idiopathic, isolated GHD (I-GHD) [To make the diagnosis of I-GHD in a short child, the National Institute for Health and Care Excellence (NICE) recommends at least two GH stimulation tests. These tests measure the peak GH concentration in the blood following an injection with a stimulating substance such as glucagon or insulin, and I-GHD is diagnosed if both show a peak GH < 6.7 μg/L [formerly 20 mU/L] [A number of studies have shown that sex steroid priming improves the response to provocative testing [Although I-GHD is known to reverse in many children, traditional practice is to continue treatment with daily injections of GH until FH is achieved. Establishing normal GH status in early puberty would relieve patients from the diagnostic uncertainty of GHD persistence and may allow patients to stop GH therapy earlier whilst still reaching a normal FH. This would relieve them of the unpleasant and inconvenient burden of daily injections of an unnecessary medication and considerably reduce health care cost [This phase III, international, multi-centre, open label, randomised, controlled, non-inferiority trial will assess the safety, efficacy, health-related quality of life, cost effectiveness, biochemical and bone health effects of discontinuing GH therapy in children who have a normal GH re-test in established puberty. The acceptability of the trial and treatment pathways to patients, parents and staff will be explored via a qualitative research sub-study.