Cargando…

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

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, genet...

Descripción completa

Detalles Bibliográficos
Autores principales: Wit, Jan M., Deeb, Asma, Bin-Abbas, Bassam, Al Mutair, Angham, Koledova, Ekaterina, Savage, Martin O.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Galenos Publishing 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6878339/
https://www.ncbi.nlm.nih.gov/pubmed/31284701
http://dx.doi.org/10.4274/jcrpe.galenos.2019.2019.0088
_version_ 1783473477974491136
author Wit, Jan M.
Deeb, Asma
Bin-Abbas, Bassam
Al Mutair, Angham
Koledova, Ekaterina
Savage, Martin O.
author_facet Wit, Jan M.
Deeb, Asma
Bin-Abbas, Bassam
Al Mutair, Angham
Koledova, Ekaterina
Savage, Martin O.
author_sort Wit, Jan M.
collection PubMed
description 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.
format Online
Article
Text
id pubmed-6878339
institution National Center for Biotechnology Information
language English
publishDate 2019
publisher Galenos Publishing
record_format MEDLINE/PubMed
spelling pubmed-68783392019-12-04 Achieving Optimal Short- and Long-term Responses to Paediatric Growth Hormone Therapy Wit, Jan M. Deeb, Asma Bin-Abbas, Bassam Al Mutair, Angham Koledova, Ekaterina Savage, Martin O. J Clin Res Pediatr Endocrinol Review 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. Galenos Publishing 2019-12 2019-11-22 /pmc/articles/PMC6878339/ /pubmed/31284701 http://dx.doi.org/10.4274/jcrpe.galenos.2019.2019.0088 Text en ©Copyright 2019 by Turkish Pediatric Endocrinology and Diabetes Society | The Journal of Clinical Research in Pediatric Endocrinology published by Galenos Publishing House. http://creativecommons.org/licenses/by/2.5/ This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Review
Wit, Jan M.
Deeb, Asma
Bin-Abbas, Bassam
Al Mutair, Angham
Koledova, Ekaterina
Savage, Martin O.
Achieving Optimal Short- and Long-term Responses to Paediatric Growth Hormone Therapy
title Achieving Optimal Short- and Long-term Responses to Paediatric Growth Hormone Therapy
title_full Achieving Optimal Short- and Long-term Responses to Paediatric Growth Hormone Therapy
title_fullStr Achieving Optimal Short- and Long-term Responses to Paediatric Growth Hormone Therapy
title_full_unstemmed Achieving Optimal Short- and Long-term Responses to Paediatric Growth Hormone Therapy
title_short Achieving Optimal Short- and Long-term Responses to Paediatric Growth Hormone Therapy
title_sort achieving optimal short- and long-term responses to paediatric growth hormone therapy
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6878339/
https://www.ncbi.nlm.nih.gov/pubmed/31284701
http://dx.doi.org/10.4274/jcrpe.galenos.2019.2019.0088
work_keys_str_mv AT witjanm achievingoptimalshortandlongtermresponsestopaediatricgrowthhormonetherapy
AT deebasma achievingoptimalshortandlongtermresponsestopaediatricgrowthhormonetherapy
AT binabbasbassam achievingoptimalshortandlongtermresponsestopaediatricgrowthhormonetherapy
AT almutairangham achievingoptimalshortandlongtermresponsestopaediatricgrowthhormonetherapy
AT koledovaekaterina achievingoptimalshortandlongtermresponsestopaediatricgrowthhormonetherapy
AT savagemartino achievingoptimalshortandlongtermresponsestopaediatricgrowthhormonetherapy