Approach to the Patient: Safety of Growth Hormone Replacement in Children and Adolescents.

Recombinant human growth hormone (rhGH) was initially approved in 1985 for severe GH deficiency and has since expanded to treat several conditions associated with poor growth and short stature.

• Initial approval was specifically for patients with severe GH deficiency (GHD) in 1985.
• rhGH is now approved for multiple conditions beyond severe GHD.
• The expanded use has occurred over more than 3 decades of clinical application.

Recent studies have raised concerns that rhGH treatment during childhood may affect morbidity and mortality in adulthood.

• Specific controversies identified include cancer risk and cerebrovascular events.
• Both short- and long-term effects of rhGH beyond height outcomes were reviewed.
• The paper addresses these concerns in the context of common pediatric endocrinology referrals.

The paper reviewed three common clinical referral scenarios to a pediatric endocrinology clinic to contextualize rhGH safety counseling.

• Each clinical case was used to highlight differences in counseling and clinical outcomes.
• Methods to mitigate risk were reviewed in the context of each case.
• The case-based approach was used to apply safety data to real-world clinical scenarios.

Overall safety data for rhGH therapy in children and adolescents are described as largely reassuring.

• The review covered both short- and long-term effects of rhGH beyond height outcomes.
• Data accumulated over more than 3 decades of use inform the safety profile.
• Despite reassuring data, the authors note that much remains to be learned about pharmaceutical approaches to growth in children and the lifelong effect of treatment.