Current Indications for Growth Hormone Therapy ( Endocrine Development )

Publication series : Endocrine Development

Author: Hindmarsh P.C.  

Publisher: S. Karger AG‎

Publication year: 2010

E-ISBN: 9783805591959

P-ISBN(Paperback): 9783805591942

Subject: R977.1 hormone drugs

Keyword: 骨科学(运动系疾病、矫形外科学),儿科学,内分泌腺疾病及代谢病

Language: ENG

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Description

Growth hormone (GH) has been used therapeutically for over 50 years. Since the development of a nearly unlimited supply of recombinant human GH in the mid-1980s, children with less severe GH deficiency can also profit from GH replacement therapy. Careful and accurate diagnosis and specific dosing, both essential to ensure normal height development, require the clinician to understand the finer points of clinical trials, to acquire quality evidence and assess the benefits of therapeutic intervention. Furthermore, genetic and environmental factors influencing GH sensitivity and responsiveness need to be taken into account. In this second edition all these aspects are covered in depth. Clinical examination, detailed auxological measurements, bone age assessment, molecular analysis and neuro-radiological evaluation as well as an adaptive strategy of dosing focusing on a patient‘s individual responsiveness are discussed in detail. This volume of Endocrine Development is essential reading for pediatric endocrinologists, pediatricians and clinical nurse specialists involved in GH therapy.

Chapter

Chapter 1: Clinical Trials: Planning and Analysis

Abstract

Introduction

Statistical Structure for Planning of Trials

Randomization

Outcomes in Studies of Growth Hormone Therapy

Longitudinal Measurements

Multiple Outcomes

Issues Related to Trial Design

Interpretation of Negative Trials

Non-Inferiority Trials

Conclusion

References

Chapter 2: The Evidence Base for Growth Hormone Effectiveness in Children

Abstract

Evidence Is Not Truth – How Do We Know What We Know?

GH Therapy – How Do We Define Benefit and What Is the Evidence?

Height Screening/Monitoring

Bone Age and Predicted Height as a Surrogate

Where Does Evidence-Based Child Health Fit in Endocrinology

Conclusions

References

Chaper 3: Safety of Recombinant Human GrowthHormone

Abstract

Short-Term Safety Issues

Longer Term Safety Considerations

Tumour Risk and Growth Hormone Treatment

Conclusion

References

Chapter 4: Diagnosis of Growth Hormone Deficiency

Abstract

Introduction

Clinical Examination and Auxology

Measuring GH Secretion

Using Downstream Targets of GH Action – IGF-1 and IGFBP-3

Neuroradiology

Conclusions

References

Chapter 5: Biological Determinants of Responsiveness to Growth Hormone: Pharmacogenomics and Personalized Medicine

Abstract

Introduction

Pharmacogenomics of Growth

GH Insensitivity (GHI)

GH Receptor

Altered GHR Signaling

Growth Responsiveness to GH Therapy, the Impact of the Growth Plate

Conclusions

References

Chapter 6: Clinical Considerations in Using Growth Hormone Therapy in Growth Hormone Deficiency

Abstract

Introduction

Growth Hormone Therapy

Conclusions

References

Chapter 7: Current Indications for Growth Hormone Therapy for Children and Adolescents

Abstract

Introduction

Growth Hormone Deficiency (GHD)

Chronic Renal Insufficiency (CRI), also Known as Chronic Kidney Disease (CKD)

Turner Syndrome (TS)

Children Born Small-for-Gestational Age (SGA), Who Fail to Catch Up to the Normal Growth Channels

Prader-Willi Syndrome (PWS)

Idiopathic Short Stature (ISS)

SHOX Gene Haploinsufficiency (SHOX)

Noonan Syndrome (NS)

Conclusions

References

Chapter 8: GH Use in the Transition of Adolescence to Adulthood

Abstract

Introduction

Who, When and How Do We Retest for GH Deficiency during Transition?

Efficacy Data

Dose

Follow-Up

Conclusions

References

Author Index

Subject Index

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