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Growth problems and growth hormone treatment in children with cystic fibrosis
1Department of Pediatrics, University of Texas Southwestern Medical School, Dallas 75390-9063, USA. dana.hardin@utsouthwestern.edu
Insights
Growth hormone (GH) therapy shows promise for improving linear growth and weight gain in children with cystic fibrosis (CF). Early findings suggest better clinical outcomes, including improved lung function and fewer hospitalizations.
Area of Science:
- Pediatric Endocrinology
- Pulmonology
- Genetics
Background:
- Children with cystic fibrosis (CF) often experience impaired linear growth and weight gain despite nutritional support.
- Growth velocity can be significantly reduced during puberty in CF patients, irrespective of clinical condition or sex hormones.
Purpose of the Study:
- To evaluate the potential benefits of growth hormone (GH) treatment in improving growth parameters in children with CF.
- To explore the impact of GH therapy on clinical status, including pulmonary function and hospitalization rates.
Main Methods:
- Review of existing studies and clinical observations regarding GH treatment in pediatric CF patients.
- Ongoing large multicenter trial assessing long-term GH therapy effects on quality of life and pulmonary status.
Main Results:
- Previous studies suggest GH treatment significantly enhances height velocity and weight gain in children with CF.
- Clinical experience indicates improved growth is linked to reduced hospitalizations, antibiotic use, and better pulmonary function.
Conclusions:
- Growth hormone therapy presents a potential therapeutic strategy for addressing growth deficits in pediatric cystic fibrosis.
- Further investigation through ongoing trials is crucial to confirm long-term benefits on quality of life and pulmonary health.
Abstract:
Children with cystic fibrosis (CF) typically demonstrate poor linear growth and, despite aggressive nutritional supplementation, suboptimal weight gain. Growth velocity may be particularly blunted during puberty, a finding that occurs independently of clinical status or sex steroid levels. Several studies indicate that growth hormone (GH) treatment may be associated with significant improvement in both height velocity and weight gain in children with CF. These findings may translate into improvements in clinical status. In our experience, improved growth and weight status have been associated with fewer hospitalizations, fewer antibiotic courses, and improved pulmonary function. A large multicenter trial is currently underway to assess the effect of long-term therapy with GH on the quality of life and pulmonary status.