Safety of Pediatric rhGH Therapy: An Overview and the Need for Long-Term Surveillance

Area of Science:

• Pediatric Endocrinology
• Long-term Health Outcomes
• Pharmacovigilance

Background:

• Growth hormone (GH) therapy, initiated in 1958, has a generally good short-term safety record.
• Concerns persist regarding potential long-term adverse effects in individuals treated during childhood.
• Recent observational studies present conflicting data on the safety of GH therapy.

Purpose of the Study:

• To provide a comprehensive review of studies assessing the long-term safety of recombinant human growth hormone (rhGH) therapy.
• To evaluate the evidence concerning the risks of cancer, cardiovascular diseases, and diabetes in patients treated with rhGH during childhood.
• To synthesize current knowledge on the long-term safety profile of rhGH therapy.

Main Methods:

• Systematic review of observational studies.
• Analysis of data from cohorts of young adult patients treated with GH in childhood.
• Focus on studies reporting on cancer incidence, cardiovascular events, and diabetes development.

Main Results:

• Conflicting results from various cohorts regarding long-term safety.
• Evidence for and against an increased risk of cancer in rhGH-treated individuals.
• Inconclusive findings on the association between rhGH therapy and cardio/cerebrovascular diseases or diabetes.

Conclusions:

• The long-term safety of childhood GH therapy requires ongoing investigation.
• Further research is needed to clarify the risks of cancer, cardiovascular diseases, and diabetes.
• Conflicting data highlights the complexity of assessing long-term outcomes of rhGH treatment.