Related Experiment Videos
Safety of recombinant deoxyribonucleic acid-derived growth hormone: The National Cooperative Growth Study experience
S L Blethen1, D B Allen, D Graves
1Department of Pediatrics, State University of New York, Stony Brook 11794-8111, USA.
Insights
Recombinant human growth hormone (rhGH) treatment is generally safe for children, with rare major adverse events. However, children with renal disease face higher risks of intracranial hypertension with rhGH therapy.
Area of Science:
- Pediatric Endocrinology
- Pharmacovigilance
- Biologics Safety
Background:
- The National Cooperative Growth Study has tracked recombinant human growth hormone (rhGH) safety since 1985.
- Extensive data from over 19,000 children (47,000+ patient-years) inform safety profiles.
Purpose of the Study:
- To assess the long-term safety and adverse event profile of rhGH treatment in a large pediatric cohort.
- To identify specific patient groups with altered risk profiles for adverse events during rhGH therapy.
Main Methods:
- Longitudinal safety monitoring of pediatric patients receiving rhGH therapy.
- Statistical analysis of adverse event data, comparing risk factors and outcomes.
Main Results:
- Children with renal disease showed increased intracranial hypertension risk (P < 0.01).
- Idiopathic short stature patients had lower slipped capital femoral epiphysis risk (P < 0.01).
- No increased risk for leukemia or CNS tumor recurrence; rare antibody development (0.04%).
Conclusions:
- rhGH treatment demonstrates a favorable safety profile with rare major adverse events.
- Preexisting conditions, like renal insufficiency, can influence adverse event frequency.
- Risk stratification is crucial for optimizing rhGH therapy safety in pediatric populations.
Abstract:
The National Cooperative Growth Study has monitored the safety of recombinant human GH (rhGH) since 1985. Data have been collected from more than 19,000 children representing over 47,000 patient-years of rhGH treatment. Children receiving GH for renal disease were more likely to develop problems such as intracranial hypertension than those with GH deficiency (P < 0.01). Children with idiopathic short stature were less likely to develop slipped capital femoral epiphysis than those with GH deficiency or Turner's syndrome (P < 0.01). There was no evidence of an increased recurrence of leukemia or central nervous system tumors. There were 3 new cases of leukemia in children without known risk factors for developing leukemia and 5 cases in children with known risk factors. Growth deceleration associated with high affinity, high capacity antibodies to GH was found in only 2 of 5039 subjects tested (0.04%). Major adverse events in association with rhGH treatment have been rare, and preexisting medical conditions such as renal insufficiency may affect their frequency.