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GH and IGF-1 Replacement in Children

  • Roland PfäffleEmail author
  • Wieland Kiess
Chapter
Part of the Handbook of Experimental Pharmacology book series

Abstract

In this chapter, we want to give an overview on what we have learned from more than 30 years ago on the use of recombinant human growth hormone (rhGH) and later recombinant human IGF-1 which was introduced for the treatment of short children and what are the safety issues concerned with this treatment. However, rhGH is used not solely in conditions where short stature is the consequence of GH deficiency but also in various disorders without a proven GH deficiency. In clinical studies, growth responses to various forms of rhGH therapy were analyzed, adding to our concept about the physiology of growth. Most patients under rhGH treatment show a considerable short-term effect; however, the long-term gain of height in a child obtained by a year-long treatment until final height remains controversial in some of the growth disorders that have been treated with rhGH or IGF-1. Today the first studies on the long-term safety of rhGH treatment have been published and raising some questions whether this treatment is similarly safe for all the patient groups treated with rhGH. Although there is a long-standing safety record for these hormone replacement therapies, in the face of the considerable costs involved, the discussion about the risk to benefit ratio is continuing. Newer developments of rhGH treatment include long-term preparations, which have only to be injected once a week. Although some of these drugs already have proven their non-inferiority to conventional rhGH treatment, we have to await further results to see whether they show improvements in treatment adherence of the patients and prove their long-term safety.

Keywords

Growth hormone Growth hormone deficiency Idiopathic short stature Insulin-like growth factor I Long-acting growth hormone Noonan syndrome Prader-Willi syndrome Short stature Small for gestational age Turner syndrome 

Abbreviations

CJD

Creutzfeldt-Jakob disease

EMA

European Medicines Agency

FDA

Food and Drug Administration

GH

Growth hormone, somatropin

GHD

GH deficiency

GHIS

Growth hormone insensitivity syndrome

GHR

Growth hormone receptor

HT

Height

HV

Height velocity

IGFBP-3

Insulin-like growth factor binding protein 3

IGF-I

Insulin-like growth factor 1

IGHD

Isolated growth hormone deficiency

ISS

Idiopathic short stature

NS

Noonan syndrome

PEGylated

(Covalently) bound to polyethylene glycol

PIGFD

Primary insulin-like growth factor 1 deficiency

PTPN11

Protein tyrosine phosphatase, nonreceptor type 11

PWS

Prader-Willi syndrome

rhGH

Recombinant human growth hormone

SDS

Standard deviation (SD) score

SGA

Small for gestational age

SHOX

Short stature homeobox-containing gene

SIR

Standard incidence ratio

SMR

Standard mortality ratio

TS

Turner syndrome

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Authors and Affiliations

  1. 1.Department of Pediatric EndocrinologyUniversity Children’s Hospital LeipzigLeipzigGermany

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