Long-Acting Growth Hormone Therapy in Pediatric Growth Hormone Deficiency: A Consensus Statement

Area of Science:

• Pediatric Endocrinology
• Pharmacology
• Biotechnology

Background:

• Limited guidance exists for using new long-acting growth hormone (LAGH) therapies in children with growth hormone (GH) deficiency.
• Recent advancements have introduced LAGH formulations, necessitating updated clinical practice recommendations.

Purpose of the Study:

• To establish an international consensus on the clinical use of LAGH in pediatric GH deficiency.
• To provide guidance for healthcare practitioners on selecting and managing LAGH therapies for children.

Main Methods:

• An international consensus group of 11 pediatric endocrinology experts from 10 countries was convened.
• Recommendations were developed through online and in-person meetings, supported by a targeted literature review.
• Formulations were selected based on international phase III trials and multi-country regulatory approvals.

Main Results:

• LAGH may reduce injection frequency and treatment burden, potentially improving adherence and outcomes in pediatric GH deficiency.
• Key topics addressed include patient selection, dosing, therapy initiation/switching, administration, and adherence.
• Current data on LAGH in this population are primarily from clinical trials, with a lack of long-term, real-world evidence.

Conclusions:

• This consensus provides practical guidance for using LAGH in children with GH deficiency.
• Further long-term, real-world data are essential to refine evidence-based recommendations for LAGH therapy.