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Achieving Optimal Short- and Long-term Responses to Paediatric Growth Hormone Therapy
Jan M. Wit1, Asma Deeb2, Bassam Bin-Abbas3
1Leiden University Medical Centre, Department of Paediatrics, Leiden, Netherlands
Insights
Human growth hormone (GH) therapy has advanced significantly over 60 years. Modern genetic diagnostics and individualized dosing improve treatment for short stature, enhancing growth outcomes.
Area of Science:
- Pediatrics
- Endocrinology
- Genetics
Background:
- Human growth hormone (GH) therapy has been used for over sixty years.
- Recombinant human GH has been available for over thirty years for GH deficiency and other disorders.
- Advances in genetic analysis have improved the diagnosis of short stature causes.
Purpose of the Study:
- To review the evidence of GH therapy efficacy in various conditions.
- To discuss the evolution of GH dosing strategies.
- To highlight the importance of assessing growth response and managing suboptimal outcomes.
Main Methods:
- Review of evidence for GH efficacy.
- Discussion of diagnostic advances using genetic analysis (Sanger, next-generation sequencing, whole exome sequencing).
- Analysis of evolving GH dosing strategies from standard to individualized.
Main Results:
- Genetic discoveries have elucidated various causes of short stature, enabling targeted therapy.
- Individualized GH dosing, based on diagnosis and growth predictors, has evolved from standard protocols.
- Evidence supports GH efficacy in GH deficiency, Turner syndrome, and SGA individuals.
Conclusions:
- Genetic diagnostics have refined the understanding and treatment of short stature.
- Optimizing GH therapy involves individualized dosing and critical assessment of growth response.
- Managing poor growth response and ensuring adherence are crucial for successful treatment outcomes.
Abstract:
It is over sixty years since the first administration of human growth hormone (GH) to children with GH deficiency, and over thirty years since recombinant human GH has been available for treatment of GH deficiency and a wider range of non-GH deficiency disorders. From a diagnostic perspective, genetic analysis, using single gene or Sanger sequencing and more recently next generation or whole exome sequencing, has brought advances in the diagnosis of specific causes of short stature, which has enabled therapy to be targeted more accurately. Genetic discoveries have ranged from defects of pituitary development and GH action to abnormalities in intracellular mechanisms, paracrine regulation and cartilage matrix formation. The strategy of GH therapy using standard doses has evolved to individualised GH dosing, depending on diagnosis and predictors of growth response. Evidence of efficacy of GH in GH deficiency, Turner syndrome and short children born small for gestational age is reviewed. The importance of critical assessment of growth response is discussed, together with the recognition and management of a poor or unsatisfactory growth response and the organisational issues related to prevention, detection and intervention regarding suboptimal adherence to GH therapy.
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