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J Clin Res Pediatr Endocrinol. 2019 Nov 22;11(4):329-340. doi: 10.4274/jcrpe.galenos.2019.2019.0088. Epub 2019 Jul 9.

Achieving Optimal Short- and Long-term Responses to Paediatric Growth Hormone Therapy

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Leiden University Medical Centre, Department of Paediatrics, Leiden, Netherlands
Mafraq Hospital, Clinic of Paediatric Endocrinology, Abu Dhabi, United Arab Emirates
King Faisal Specialist Hospital and Research Center, Department of Paediatrics, Riyadh, Saudi Arabia
King Abdullah Specialised Children’s Hospital, Ministry of National Guard Health Affairs, Riyadh, Saudi Arabia
King Abdullah International Medical Research, Clinic of Paediatrics, Riyadh, Saudi Arabia
King Saud bin Abdulaziz University for Health Science, Clinic of Paediatrics, Riyadh, Saudi Arabia
Global Medical Affairs Endocrinology, Merck KGaA, Darmstadt, Germany
William Harvey Research Institute, Barts and the London Faculty of Medicine and Dentistry, London, United Kingdom


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.


short stature; growth hormone therapy; growth hormone deficiency; Turner syndrome; small for gestational age; Paediatrics

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