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Published on: January 7, 2016
The basis for optimising growth with growth hormone usage in children with idiopathic short stature: analysis of data
Michael B Ranke1, Anders Lindberg
1University Children's Hospital, Universitätsklinikum Tübingen, Tübingen, Germany.
Insights
Growth hormone (GH) treatment can improve adult height in children with idiopathic short stature (ISS). This study identifies which ISS children benefit most from GH therapy for optimal height outcomes.
Area of Science:
- Pediatric Endocrinology
- Growth Hormone Therapy
- Short Stature Research
Background:
- Idiopathic short stature (ISS) often results in short adult height.
- Growth hormone (GH) treatment is approved in the US for ISS but debated in Europe.
- Existing data on GH efficacy in ISS is extensive but requires further categorization.
Purpose of the Study:
- To identify children with ISS most likely to achieve normal adult height with GH treatment.
- To determine which ISS patients gain the most height from GH therapy.
- To provide a basis for discussing effective and cost-efficient GH use in pediatric short stature.
Main Methods:
- Analysis of published literature on GH treatment for ISS.
- Review of data from the Pfizer International Growth Study Database (KIGS).
- Examination of a KIGS survey of 454 patients who reached adult height.
Main Results:
- Categorization of ISS children based on their potential for height gain with GH treatment.
- Identification of patient subgroups with the greatest likelihood of achieving normal adult height.
- Evidence supporting the efficacy of GH treatment in specific ISS populations.
Conclusions:
- GH treatment offers a viable option for improving adult height in select children with ISS.
- Further research and data analysis are crucial for optimizing GH treatment strategies.
- Evidence-based guidelines can enhance the efficacy and cost-effectiveness of GH therapy for pediatric short stature.
Abstract:
Many children with idiopathic short stature (ISS) remain short as adults. Height can be improved permanently by growth hormone (GH) treatment, as in children with Turner syndrome or born small for gestational age. Although GH treatment for ISS is an approved indication in the United States, there is ongoing controversy in Europe about approving GH treatment for this indication as well as determining the optimal mode of treatment. In addition to data from randomized controlled trials, data from pharmacoepidemiological surveys such as the Pfizer International Growth Study Database (KIGS) have provided substantial information about the short-term and long-term effects of GH on growth and adult height in children with ISS. Based on published articles and a recent survey from KIGS on 454 patients who have reached adult height, we attempted to categorize those children with ISS who have the greatest chances of achieving a normal adult height, or at least gain the most height following GH treatment. This analysis provides a rational basis for discussion about the most efficacious and cost-effective uses of GH treatment in children with ISS.
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