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Growth hormone treatment for idiopathic short stature

Author
Alan D Rogol, MD, PhD
Section Editor
Mitchell E Geffner, MD
Deputy Editor
Alison G Hoppin, MD

INTRODUCTION

Treatment of children and adolescents with growth hormone for idiopathic short stature (ISS) is controversial for two reasons. First, the response to growth hormone is highly variable, probably because of the heterogeneous endocrinologic profile in children with ISS, and those children who respond to growth hormone treatment may have only modest increases in linear growth. Second, there is little evidence that short stature represents a substantial psychosocial burden to most short children. The possible psychosocial benefit must be weighed against the substantial cost and possible adverse effects of treatment.

The efficacy and potential risks of growth hormone and alternative treatments for children with ISS are discussed below. The clinical evaluation of children with short stature is discussed separately. (See "Diagnostic approach to children and adolescents with short stature" and "Causes of short stature".)

CLINICAL FEATURES

Definition — Idiopathic short stature (ISS) is a clinical description rather than a disease. A practical definition of ISS is a height below 2 standard deviations (SD) of the mean for age (ie, below the 2.3rd percentile), in the absence of any endocrine, metabolic, or other disease that explains the short stature [1,2]. In defining ISS for the indication of growth hormone treatment, the US Food and Drug Administration (FDA) uses the more stringent criterion of 2.25 SD below the mean (ie, the 1.2nd percentile), and a predicted adult height that is below the normal range; this corresponds to an adult height <63 inches for males and <59 inches for females. The clinical approach to diagnosis of ISS is discussed separately. (See "Diagnostic approach to children and adolescents with short stature".)  

ISS is generally considered a normal variant of growth (since pathological causes are excluded, by definition). Other normal variants are familial short stature (FSS) and constitutional delay of growth and puberty (CDGP), sometimes called constitutional short stature for prepubertal children. There is ongoing controversy about the nomenclature of ISS. We use the term to refer to nonfamilial cases, but some other experts consider FSS and CDGP to be subcategories of ISS [1,3]. In either case, it is clear that there is considerable overlap between these variants, and many healthy children with short stature have growth characteristics consistent with more than one of these categories. The term ISS is generally reserved for children at the more severe end of the spectrum in each category, but the distinction between normal and abnormal growth patterns is not clear. (See "Causes of short stature", section on 'Normal variants of growth'.)  

Endocrine findings — Children with ISS often have normal growth velocity (often near or at the lower limit of normal), no biochemical or other evidence for a specific growth-restricting condition, and have normal results of growth hormone stimulation tests. Thus, children with ISS do not have growth hormone deficiency according to the classic criteria.  

                      

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Literature review current through: Nov 2016. | This topic last updated: Thu Mar 24 00:00:00 GMT 2016.
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