Pharmacological interventions for short stature

Pros and cons

Ronald (Ron) Rosenfeld

Research output: Contribution to journalArticle

5 Citations (Scopus)

Abstract

Although growth hormone (GH) therapy is virtually always effective in accelerating growth and restoring height potential to children with GH deficiency (GHD), the expansion of its use to a wide variety of other clinical disorders associated with short stature has resulted in considerable ethical and cost-benefit issues. Logic would demand that therapy should either be restricted to true 'replacement', thereby limiting its use to cases of unequivocal GHD, or treatment should be considered as a legitimate 'enhancement', and be available to all children with significant short stature. Consideration of the latter option requires a careful look at issues surrounding efficacy (both in terms of stature and any perceived disability resulting therefrom), cost and potential adverse effects. Similar concerns involve treatment with insulin-like growth factor-I and any related growth-augmenting therapy. To date, safety issues have been addressed through pharmaceutical-sponsored postmarketing surveillance studies. While of definite use, such investigations also have significant limitations, especially in addressing long-term concerns. The possibility of lifespan cohort studies, with surveillance of all GH recipients throughout life and comparison with data from appropriate controls, should be considered.

Original languageEnglish (US)
Pages (from-to)207-217
Number of pages11
JournalNestle Nutrition Institute Workshop Series
Volume71
DOIs
StatePublished - 2013

Fingerprint

Pharmacology
somatotropin
Growth Hormone
therapeutics
monitoring
Therapeutics
insulin-like growth factor I
cohort studies
Growth
Insulin-Like Growth Factor I
adverse effects
Cost-Benefit Analysis
Cohort Studies
drugs
Safety
Costs and Cost Analysis
Pharmaceutical Preparations

ASJC Scopus subject areas

  • Food Science
  • Pediatrics, Perinatology, and Child Health
  • Nutrition and Dietetics
  • Medicine(all)

Cite this

Pharmacological interventions for short stature : Pros and cons. / Rosenfeld, Ronald (Ron).

In: Nestle Nutrition Institute Workshop Series, Vol. 71, 2013, p. 207-217.

Research output: Contribution to journalArticle

@article{ea782947b34644f0a50cf87517bcf7ba,
title = "Pharmacological interventions for short stature: Pros and cons",
abstract = "Although growth hormone (GH) therapy is virtually always effective in accelerating growth and restoring height potential to children with GH deficiency (GHD), the expansion of its use to a wide variety of other clinical disorders associated with short stature has resulted in considerable ethical and cost-benefit issues. Logic would demand that therapy should either be restricted to true 'replacement', thereby limiting its use to cases of unequivocal GHD, or treatment should be considered as a legitimate 'enhancement', and be available to all children with significant short stature. Consideration of the latter option requires a careful look at issues surrounding efficacy (both in terms of stature and any perceived disability resulting therefrom), cost and potential adverse effects. Similar concerns involve treatment with insulin-like growth factor-I and any related growth-augmenting therapy. To date, safety issues have been addressed through pharmaceutical-sponsored postmarketing surveillance studies. While of definite use, such investigations also have significant limitations, especially in addressing long-term concerns. The possibility of lifespan cohort studies, with surveillance of all GH recipients throughout life and comparison with data from appropriate controls, should be considered.",
author = "Rosenfeld, {Ronald (Ron)}",
year = "2013",
doi = "10.1159/000342640",
language = "English (US)",
volume = "71",
pages = "207--217",
journal = "Nestl{\'e} Nutrition Institute workshop series",
issn = "1664-2155",
publisher = "S. Karger AG",

}

TY - JOUR

T1 - Pharmacological interventions for short stature

T2 - Pros and cons

AU - Rosenfeld, Ronald (Ron)

PY - 2013

Y1 - 2013

N2 - Although growth hormone (GH) therapy is virtually always effective in accelerating growth and restoring height potential to children with GH deficiency (GHD), the expansion of its use to a wide variety of other clinical disorders associated with short stature has resulted in considerable ethical and cost-benefit issues. Logic would demand that therapy should either be restricted to true 'replacement', thereby limiting its use to cases of unequivocal GHD, or treatment should be considered as a legitimate 'enhancement', and be available to all children with significant short stature. Consideration of the latter option requires a careful look at issues surrounding efficacy (both in terms of stature and any perceived disability resulting therefrom), cost and potential adverse effects. Similar concerns involve treatment with insulin-like growth factor-I and any related growth-augmenting therapy. To date, safety issues have been addressed through pharmaceutical-sponsored postmarketing surveillance studies. While of definite use, such investigations also have significant limitations, especially in addressing long-term concerns. The possibility of lifespan cohort studies, with surveillance of all GH recipients throughout life and comparison with data from appropriate controls, should be considered.

AB - Although growth hormone (GH) therapy is virtually always effective in accelerating growth and restoring height potential to children with GH deficiency (GHD), the expansion of its use to a wide variety of other clinical disorders associated with short stature has resulted in considerable ethical and cost-benefit issues. Logic would demand that therapy should either be restricted to true 'replacement', thereby limiting its use to cases of unequivocal GHD, or treatment should be considered as a legitimate 'enhancement', and be available to all children with significant short stature. Consideration of the latter option requires a careful look at issues surrounding efficacy (both in terms of stature and any perceived disability resulting therefrom), cost and potential adverse effects. Similar concerns involve treatment with insulin-like growth factor-I and any related growth-augmenting therapy. To date, safety issues have been addressed through pharmaceutical-sponsored postmarketing surveillance studies. While of definite use, such investigations also have significant limitations, especially in addressing long-term concerns. The possibility of lifespan cohort studies, with surveillance of all GH recipients throughout life and comparison with data from appropriate controls, should be considered.

UR - http://www.scopus.com/inward/record.url?scp=84879256995&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84879256995&partnerID=8YFLogxK

U2 - 10.1159/000342640

DO - 10.1159/000342640

M3 - Article

VL - 71

SP - 207

EP - 217

JO - Nestlé Nutrition Institute workshop series

JF - Nestlé Nutrition Institute workshop series

SN - 1664-2155

ER -