Trends In Reported Outcomes for Growth Hormone Therapy In Children With Growth Hormone Deficiency

Area of Science:

• Pediatric Endocrinology
• Pharmacovigilance
• Clinical Trial Methodology

Background:

• Childhood growth hormone deficiency (GHD) is a significant endocrine disorder.
• Recombinant human growth hormone (rhGH) therapy is a primary treatment for GHD in pediatric populations.
• Understanding the spectrum of reported outcomes is crucial for optimizing rhGH therapy.

Purpose of the Study:

• To systematically review the frequency and trends of reported safety and effectiveness outcomes for rhGH therapy in children with GHD.
• To identify the most commonly assessed parameters in rhGH treatment studies.
• To inform the development of a standardized core outcome set for pediatric GHD.

Main Methods:

• A systematic review of studies published between 2003 and 2022 in English and Chinese databases.
• Inclusion criteria: participants initiating rhGH before age 16 for GHD.
• Data extraction focused on reported outcomes, study design, and participant demographics.

Main Results:

• 219 studies (171 cohort, 39 controlled trials, 9 case-control) were included.
• Most frequent outcomes: height SDS (53%), height velocity (48%), IGF-1 (30%), and injection site adverse events (20%).
• Over 50% of outcomes focused on safety, with efficacy outcomes also frequently reported; quality of life outcomes were infrequent (3%).

Conclusions:

• Height SDS and height velocity have consistently been the most reported efficacy outcomes over two decades.
• A significant proportion of reported outcomes relate to safety, including adverse events and biochemical markers.
• The findings support the development and implementation of a core outcome set for consistent evaluation of rhGH therapy in children with GHD.