Lessons from the national cooperative growth study

Area of Science:

• Pediatric Endocrinology
• Pharmacovigilance
• Growth Hormone Therapy

Background:

• The National Cooperative Growth Study (NCGS) was initiated in 1985.
• It is a national post-marketing surveillance program for biosynthetic growth hormone (GH) products.
• The program coordinates with the release of recombinant DNA biosynthetic GH.

Purpose of the Study:

• To review the National Cooperative Growth Study (NCGS).
• To summarize the extensive experience of the NCGS in children treated with biosynthetic GH.
• To present representative data on the efficacy and safety of GH products.

Main Methods:

• A multicenter, observational surveillance registry.
• Data collected from over 47,000 patients representing 165,000 patient-years.
• Utilized the GrowTrak program, a secure and user-friendly database.

Main Results:

• Demonstrated efficacy of GH therapy in various diagnoses like isolated (IGHD) and organic growth hormone deficiency (OGHD), idiopathic short stature (ISS), and Turner syndrome.
• Safety monitoring showed no association between GH therapy and leukemia, nonleukemic neoplasms, or CNS tumor recurrence.
• Identified a small risk of intracranial hypertension and slipped capital femoral epiphyses, particularly in children with renal disease or Turner syndrome.

Conclusions:

• The NCGS is a valuable tool for monitoring the safety and efficacy of biosynthetic GH.
• Wide physician participation was achieved through user-friendly data collection software and data sharing.
• The study provided extensive efficacy and safety data on biosynthetic GH products.