Growth hormone & IGF research : official journal of the Growth Hormone Research Society and the International IGF Research Society · 2025
Efficacy, safety, and outcomes of growth hormone treatment in children with idiopathic short stature.
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Linglart Agnès, Dauber Andrew, de Lima Jorge Alexander, Luo Xiaoping, Ogata Tsutomu, Sävendahl Lars
Аннотация
BACKGROUND: Growth hormone (GH) is a treatment option in some countries for children with idiopathic short stature (ISS) given to enable them to attain height within the expected range. Currently, it is not often utilised in clinical practice. A literature review was conducted to summarise the efficacy, safety, and outcomes associated with GH treatment in children with ISS.
SUMMARY: Guidelines for the diagnosis and treatment of ISS may benefit from revision to accommodate recent findings on genetic factors that influence height. Clinical trials and observational studies designed to investigate the effect of GH treatment on children with ISS have shown that it is effective in enabling them to attain height within the normal range. In some instances, height improvements are reported up to adult height. The safety of GH treatment in this patient population has also been investigated and no new safety concerns have been observed. In analyses that were designed to investigate the effect of GH on quality of life, improvements in psychosocial scores were observed, either by the patient, parent, or the treating physician.
KEY MESSAGE: GH treatment is effective in improving height outcomes and quality of life in children with ISS, with an acceptable safety profile.
SUMMARY: Guidelines for the diagnosis and treatment of ISS may benefit from revision to accommodate recent findings on genetic factors that influence height. Clinical trials and observational studies designed to investigate the effect of GH treatment on children with ISS have shown that it is effective in enabling them to attain height within the normal range. In some instances, height improvements are reported up to adult height. The safety of GH treatment in this patient population has also been investigated and no new safety concerns have been observed. In analyses that were designed to investigate the effect of GH on quality of life, improvements in psychosocial scores were observed, either by the patient, parent, or the treating physician.
KEY MESSAGE: GH treatment is effective in improving height outcomes and quality of life in children with ISS, with an acceptable safety profile.