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Growth outcomes in individuals with idiopathic short stature treated with growth hormone therapy
1Göteborg Paediatric Growth Research Centre, Sahlgrenska Academy, Göteborg University, Göteborg, Sweden.
Insights
Growth hormone (GH) treatment significantly improves long-term height outcomes for children with idiopathic short stature (ISS). Benefits are comparable to those in GH-deficient children and maintained into adulthood.
Area of Science:
- Pediatrics
- Endocrinology
- Growth Disorders
Background:
- Idiopathic short stature (ISS) is a condition where children are significantly shorter than average without a clear medical cause.
- Evaluating long-term growth outcomes in children with ISS receiving growth hormone (GH) treatment is crucial for understanding treatment efficacy.
Purpose of the Study:
- To synthesize findings from recent studies on the long-term height outcomes of GH treatment in children with ISS.
- To assess the magnitude and influencing factors of height gain in response to GH therapy for ISS.
Main Methods:
- A literature search was conducted for randomized and nonrandomized studies over the past 10 years.
- Included studies focused on GH treatment in prepubertal children with ISS up to adult height.
- Six relevant publications were identified, with no classic meta-analyses.
Main Results:
- GH treatment showed a dose-dependent response in height outcome for children with ISS.
- Younger age and a greater height deficit at treatment initiation correlated with more substantial height gains (0.5–1.3 SDS).
- Height gains were comparable to those in GH-deficient children and other non-GH-deficient syndromes, even after controlling for spontaneous growth.
Conclusions:
- Children with ISS can achieve significant and sustained height improvement with GH treatment.
- Treatment benefits are comparable to those observed in GH-deficient populations and maintained into adulthood.
- Factors like age and initial height deficit influence the degree of height gain.
Abstract:
The few studies that have evaluated the long-term height outcomes following growth hormone (GH) treatment in children with idiopathic short stature (ISS) have shown a growth response to GH treatment similar to that in GH-deficient children. A literature search of all randomized and nonrandomized studies of GH treatment in children with ISS from prepubertal years to adult height or near-adult height published over the last 10 years identified six publications (none was a classic meta-analysis). Several studies showed a dose-dependent response in height outcome. Overall, the younger the patient and the greater the difference in current height vs. parental height at start of treatment, the more substantial the gain in height. Height improvement ranged from 0.5 to 1.3 standard deviation score (SDS). The magnitude of height gain was substantial, even after adjustment for growth changes in control subjects (0.5 to 1 SDS), and it was comparable to that seen in other non-GH-deficient syndromes. Only two studies reported data from matched control subjects. Interestingly, there was no difference in height gain between familial short stature and non-familial short stature after adjusting for spontaneous height gain to adult age in non-GH-treated control subjects. To summarize, patients with ISS can benefit from GH treatment with respect to growth outcome, with results maintained into adulthood.
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