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Short- and long-term (final height) data in children with normal variant short stature treated with growth hormone
Insights
Growth hormone (GH) treatment for children with normal variant short stature showed initial height gains but minimal final height improvement. The dose did not significantly impact outcomes, questioning its widespread use.
Area of Science:
- Pediatric Endocrinology
- Growth Hormone Therapy
- Childhood Growth Disorders
Background:
- Normal variant short stature (NVSS) affects children with predicted adult heights below -2 standard deviation scores (SDS).
- Growth hormone (GH) is a potential treatment for children with NVSS, but its efficacy and optimal dosing require further investigation.
Purpose of the Study:
- To evaluate the efficacy of different growth hormone (GH) doses in children with normal variant short stature (NVSS).
- To assess the impact of GH treatment on height prediction and final height attainment in this population.
Main Methods:
- A 5-year randomized study involving 17 children with NVSS treated with varying GH doses (3 IU/m²/day or 4.5 IU/m²/day).
- Patients were monitored for height velocity, predicted height changes (Tanner Whitehouse method), and final height attainment.
- Dose adjustments were made for one group during the study period.
Main Results:
- Significant height increases were observed in all groups after 1 and 2 years of GH treatment, irrespective of the dose.
- Height velocity decreased in the second year, and increasing GH dose did not prevent this.
- Final height was only slightly above predicted height (2.4 cm or +0.41 SDS), with final height closely matching predictions made after 1 year of therapy.
Conclusions:
- GH treatment leads to initial height prediction increases in children with NVSS, which are maintained over time.
- The overall increase in final height over predicted height is minor and may not justify the routine use of GH for NVSS.
- GH dosage did not appear to influence the observed height outcomes in this study.
Unlabelled:
Seventeen children with normal variant short stature and a predicted height below -2 SDS were treated with growth hormone (GH) six times a week for a period of 5 years. Patients were randomly selected to receive three different doses of GH, group 1 (n = 6) 3 IU/m2 per day, group 2 (n = 6) 4.5 IU/m2 per day and group 3 (n=5) 3 IU/m2 per day in the 1st year and 4.5 IU/m2 per day thereafter. There was a significant increase in height after 1 and 2 years for all patients and for all subgroups. However, this increase was not dependent on GH dose. The decrease in height velocity during the 2nd year was not prevented by the increase of GH dose in group 3. The change of predicted height after 2 years was +0.75 SDS (according to Tanner Whitehouse). Fourteen children have been treated for 4 years and 8 children for 5 years without a further change in height prediction. Nine patients have reached final height which was 2.4 cm (+0.41 SDS) above pretreatment height prediction. Final height was nearly identical to predicted height after 1 year of therapy.
Conclusion:
An increment in height prediction was observed during the first 2 years of GH treatment and maintained thereafter. However, there was only a minor increase in final height over predicted height which does not justify the general use of GH in children with normal variant short stature.