Efficacy of IGF‐based growth hormone (GH) dosing in nonGH‐deficient (nonGHD) short stature children with low IGF‐I is not related to basal IGF‐I levels. (25th January 2013)

• Record Type: Journal Article
• Authors: Cohen, Pinchas; Rogol, Alan D.; Weng, Wayne; Kappelgaard, Anne‐Marie; Rosenfeld, Ron G.; Germak, John
• Is Part Of: Clinical endocrinology. Volume 78:Number 3(2013:Mar.)
• Page Start: 405
• Page End: 414
• Publication Date: 2013-01-25
• Subjects: Endocrinology -- Periodicals; 616.4005
• DOI: 10.1111/cen.12014
• Languages: English
• ISSNs: 0300-0664

Abstract

<abstract abstract-type="main" xml:lang="en" id="cen12014-abs-0001"> <title>Summary</title> <sec id="cen12014-sec-0001" sec-type="section"> <title>Objective</title> <p>Weight‐based GH dosing is the standard for treating children with short stature. The current study validates the usefulness of IGF‐based GH dosing for GH therapy in nonGH‐deficient (nonGHD) children and its relationship with pretreatment serum IGF‐I concentration.</p> </sec> <sec id="cen12014-sec-0002" sec-type="section"> <title>Design and Patients</title> <p>In this twelve‐month, open‐label, randomized controlled study, 151 nonGHD (based on GH‐stimulation tests), prepubertal children with short stature and IGF‐I levels ≤ 33rd percentile [–0·44 standard deviation score (SDS)] were randomly assigned to receive GH (dose based on IGF‐I titration algorithm; <italic>n</italic> = 114) or to observation (<italic>n</italic> = 37). GH dose (initially 40 μg/kg/d) was adjusted every 3 months to achieve an IGF‐I SDS in the upper normal range (66–99th percentile).</p> </sec> <sec id="cen12014-sec-0003" sec-type="section"> <title>Measurements and Results</title> <p>In treated children, mean height SDS (HSDS) increased from −2·5 at baseline to −1·7 at 12 months and mean IGF‐I SDS increased from −1·7 to 0·1. These parameters remained unchanged in untreated children. There was no relationship between change in HSDS (ΔHSDS) and degree of IGF‐I deficiency at baseline. No safety problems were observed. Both groups had a similar advance in bone age. At the end of study, ΔHSDS in treated children showed a positive correlation with IGF‐I SDS, but not with GH dose [mean 59 μg/kg/d (range 29–92)], basal IGF‐I SDS or 1‐month IGF parameters.</p> </sec> <sec id="cen12014-sec-0004" sec-type="section"> <title>Conclusions</title> <p>In nonGHD subjects with short stature and serum IGF‐I concentrations within and below the lower third of normal, adjusting GH dose to achieve an IGF‐I level in the upper normal range resulted in a significant increase in HSDS, regardless of basal IGF‐I levels.</p> </sec> </abstract>