Growth Hormone Therapy in Children: Safety, Musculoskeletal Health, Precision Monitoring, and Long-Term Outcomes

Growth hormone (GH) therapy transformed the care of children with growth hormone deficiency, Turner syndrome, Prader–Willi syndrome, and children born small for gestational age, and other growth disorders. While its effectiveness in promoting linear growth is well established, clinicians increasingly recognise the successful treatment extends beyond height alone, such as metabolic balance and quality of life.

As more children receive growth hormone, and even for longer durations, and recently the long-acting growth hormone became available for certain indications, there is growing concern regarding its safety profile, especially with respect to musculoskeletal health. Reported complications include slipped capital femoral epiphysis, progression of scoliosis, joint pain, and transient bone or muscle discomfort. Although large-scale clinical trials and registries suggest that GH therapy is generally safe when appropriately managed, heterogeneity in patient response and a lack of predictive markers highlight the need for targeted risk assessment. Contemporary studies are increasingly drawing on real-world data to uncover previously under-recognized adverse patterns and to refine understanding of safety across diverse pediatric cohorts. Nonetheless, standardized frameworks for musculoskeletal monitoring, early detection of complications, and multidisciplinary preventive care remain insufficiently defined.

Despite the ongoing research, some questions remained unanswered fully, such as

- Which children are at a higher risk for adverse musculoskeletal outcomes?

- How should monitoring differ among different underlying disorders and age groups?

- What is the safety profile of long-acting growth hormone?

- Can artificial intelligence help in prediction of some of adverse outcomes?

- How does the treatment with growth hormone therapy affect the children and their families beyond the scope of the growth?

This Research Topic aims to deepen and consolidate understanding of the safety and musculoskeletal effects of GH therapy in pediatric patients by integrating evidence from clinical studies, observational registries, and real-world practice. The primary objective is to improve the identification of risk factors, enhance monitoring strategies, and optimize multidisciplinary management models that safeguard musculoskeletal health during treatment.

To gather further insights into the pediatric safety landscape of GH therapy, we welcome articles addressing, but not limited to, the following themes:

o Real-world evidence on the safety profile of GH therapy in children and adolescents

o Musculoskeletal complications: incidence, mechanisms, early detection, and management

o Risk stratification models and biomarkers for predicting adverse musculoskeletal outcomes

o The influence of GH dosage, treatment duration, and underlying aetiology on safety outcomes

o Digital health and remote monitoring

o Multidisciplinary management approaches to prevent and treat musculoskeletal side effects

o Longitudinal monitoring protocols to optimize bone and joint health

o Patient reported outlines and Quality-of-life linked to growth and musculoskeletal well-being

Appendix – Article Types

This Research Topic aims to bring together clinicians, researchers, allied health professionals, and patient advocates to explore the evolving landscape of GH therapy safety and long-term care. It welcomes diverse article types including, but not limited to:

o Original Research

o Reviews and Mini Reviews

o Systematic Reviews and Meta-Analyses

o Clinical Trial Protocols

o Case Reports

o Perspectives and Opinion Articles

Article types and fees

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Case Report
• Clinical Trial
• Editorial
• FAIR² Data
• General Commentary
• Hypothesis and Theory
• Methods
• Mini Review
• Opinion
• ... View all formats

Articles that are accepted for publication by our external editors following rigorous peer review incur a publishing fee charged to Authors, institutions, or funders.

Article types

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Case Report
• Clinical Trial
• Editorial
• FAIR² Data
• General Commentary
• Hypothesis and Theory
• Methods
• Mini Review
• Opinion
• Original Research
• Perspective
• Policy and Practice Reviews
• Review
• Study Protocol
• Systematic Review
• Technology and Code

Keywords: Growth hormone therapy, Paediatric safety, Musculoskeletal complications, Long-acting growth hormone, Growth hormone deficiency, Risk stratification, Real-world evidence, Longitudinal monitoring, Quality of life, Multidisciplinary management

Important note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.