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Anabolic effect of biosynthetic growth hormone in cystic fibrosis patients
C A Huseman1, J L Colombo, M A Brooks
1Department of Pediatrics, University of Nebraska Medical School, Omaha 68198-5180, USA.
Insights
Recombinant human growth hormone (rhGH) therapy in cystic fibrosis (CF) patients demonstrated significant anabolic effects, including improved growth velocity and nitrogen retention. While body weight did not change, patients showed increased muscle mass and decreased fat stores.
Area of Science:
- Pediatric Endocrinology
- Pulmonology
- Metabolic Disorders
Background:
- Cystic Fibrosis (CF) is a genetic disorder affecting multiple organs, often leading to growth impairment and malnutrition.
- Anabolic effects, such as improved growth and body composition, are crucial for managing CF patients.
Purpose of the Study:
- To evaluate the anabolic potential of recombinant human growth hormone (rhGH) treatment in prepubertal children with Cystic Fibrosis.
- To assess changes in growth rate, nitrogen balance, body composition, and pulmonary function following rhGH therapy.
Main Methods:
- Nine prepubertal, endocrinologically normal CF patients received rhGH (0.3 mg/kg/week) for 9-12 months.
- Measurements included growth velocity, height standard deviation scores (SDS), arm muscle area (AMA), arm fat area (AFA), nitrogen retention, somatomedin C (SmC), bone age (BA), and pulmonary function tests (PFTs).
Main Results:
- rhGH significantly increased growth velocity (5.7 to 7.8 cm/year) and height SDS.
- Patients showed increased AMA, decreased AFA, and improved nitrogen retention (positive or less negative in 5/9 patients).
- SmC levels increased significantly, and bone age advanced by 1.0 year/year; PFT improvements were not statistically significant.
Conclusions:
- Biosynthetic rhGH therapy exerts a significant anabolic effect in CF patients.
- rhGH improves growth, body composition, and nitrogen metabolism, with potential benefits for pulmonary function.
- Further research is warranted to optimize rhGH use in CF management.
Abstract:
The purpose of this study was to determine whether GH treatment of cystic fibrosis (CF) patients can result in an anabolic effect, i.e., increased weight gain, improved growth rate, nitrogen retention, and improved pulmonary function. Nine prepubertal endocrinologically normal CF patients (3 girls, 6 boys; chronological age (CA) 5.5-9.8 years, and bone age (BA) 4.5-9.0 years), received recombinant human growth hormone (rhGH) 0.3 mg/kg/week subcutaneously for a period of 12 months (N = 8) or 9 months (N = 1). Normal glucose tolerance was determined before treatment. Pulmonary function studies and anthropometric measurements were done every 3 months. Thyroid status, somatomedin C (SmC), BA, and routine chemistries were evaluated every 6 months. The pretreatment growth velocity averaged 5.7 +/- 0.3 (SE) cm/year and significantly increased to 7.8 +/- 0.4 (SE) cm/year during therapy, (P < 0.01). Standard deviation scores (SDS) for height significantly increased during rhGH therapy as compared with pretreatment, (P < 0.05). Weight of the patients during rhGH therapy did not significantly change during or after rhGH therapy. After therapy, all patients showed a significant increase in arm muscle area (AMA) and a significant decrement in arm fat area (AFA) (P < 0.01). Net nitrogen anabolism was negative in all subjects before therapy but became more positive in five patients during rhGH therapy. Three patients achieved positive nitrogen retention. SmC values significantly increased from a mean value of 0.62 +/- 0.1 (SE) U/ml to 1.6 +/- 0.6 (SE) U/ml after therapy. BA advanced 1.0 +/- 0.1 SE per year after treatment. Of the seven patients able to perform adequate pulmonary function testing, improvement occurred in FVC, FEV1.0, and PEFR in 5, 5, and 4 patients, respectively, but these changes did not reach statistical significance. We conclude that biosynthetic rhGH therapy had a significant anabolic effect in CF patients as shown by increased growth velocity, SmC values, increased protein and decreased fet stores, and a positive or less negative net nitrogen retention in five of the patients.