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Factors predicting the response to growth hormone (GH) therapy in prepubertal children with GH deficiency
S L Blethen1, P Compton, B M Lippe
1Department of Pediatrics, State University of New York, Stony Brook 11794.
Insights
Growth hormone (GH) therapy response in children with GH deficiency (GHD) depends on factors like age and GH levels. Early treatment in young children with severe idiopathic GHD offers the best outcomes.
Area of Science:
- Pediatric Endocrinology
- Growth Hormone Therapy
- Child Development
Background:
- Growth hormone deficiency (GHD) affects prepubertal children's growth.
- Understanding factors influencing GH therapy response is crucial for optimizing treatment.
- Biosynthetic methionyl GH (somatrem) is a common treatment for GHD.
Purpose of the Study:
- To identify key factors predicting the response to GH therapy in children with GHD.
- To compare treatment outcomes between idiopathic and organic GHD.
- To provide evidence-based recommendations for optimizing GH therapy.
Main Methods:
- Multiple regression analysis of data from 632 naive prepubertal children with GHD.
- Analysis of factors including age, GH levels, weight, dosing schedule, dose, and midparental height.
- Comparison of growth rates between idiopathic and organic GHD groups using analysis of covariance.
Main Results:
- In idiopathic GHD, age, log maximum GH, weight, dosing schedule, dose, and midparental height predicted 40% of treatment response.
- In organic GHD, pretreatment growth rate, log maximum GH, and age predicted 20% of response.
- Children with idiopathic GHD showed significantly better growth than those with organic GHD (9.2 vs. 8.8 cm/yr).
- Younger and thinner children responded less favorably to GH therapy.
Conclusions:
- Early diagnosis and initiation of GH therapy are vital for optimal height attainment.
- The best response to GH therapy is observed in young children with severe idiopathic GHD receiving daily, weight-adjusted doses.
- Higher daily GH doses may benefit older children with less severe or acquired GHD, or those underweight for height.
Abstract:
To identify factors influencing the response to GH therapy, we used a multiple regression model to analyze data from 632 naive prepubertal children with GH deficiency (GHD). There were 523 children with idiopathic and 109 children with organic GHD. They were treated with the same preparation of biosynthetic methionyl GH (somatrem, Protropin) for at least 1 yr. In children with idiopathic GHD, six variables predicted 40% of the response to treatment. They were (listed in relative importance, all P < 0.0001): age, log maximum GH, weight adjusted for height, dosing schedule, dose, and midparental height. Three variables, pretreatment growth rate, log maximum GH, and age, predicted 20% of the GH response in children with organic GHD. When data for all children were analyzed using analysis of covariance, children with idiopathic GHD grew better than those with organic GHD (mean +/- SD, 9.2 +/- 2.4 vs. 8.8 +/- 2.6 cm/yr; P < 0.0001). The children (both organic and idiopathic GHD) who did not respond well to treatment were younger and thinner than those who did. Early diagnosis and initiation of therapy should be beneficial to ultimate height attainment. The best response to GH therapy should be in young children with severe idiopathic GHD who receive daily weight-adjusted doses. The use of GH daily in higher doses would be expected to be most beneficial in older children with acquired and/or less severe GHD or in children who are underweight for height.
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