Management of children with idiopathic short stature

Dunkel, Leo

doi:10.1530/eje.1.02244

Cited by 5 publications

(3 citation statements)

References 26 publications

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“…In 2003, the Food and Drug Administration approved rhGH for children with ISS whose height is more than 2.25 standard deviations (SD) below the mean (or below the 1st percentile). Since the specific etiology for ISS in children is sometimes difficult to identify, children are often diagnosed with ISS and receive growth hormone (GH) therapy [ 3 , 4 ]. TS occurs ~1 in 2,000–2,300 live female births [ 5 ].…”

Section: Introductionmentioning

confidence: 99%

Switching to Omnitrope® from Other Recombinant Human Growth Hormone Therapies: A Retrospective Study in an Integrated Healthcare System

Rashid

Saenger

et al. 2014

Biol Ther

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IntroductionThis study was conducted using an integrated retrospective database to evaluate the effectiveness of Omnitrope® (Sandoz) on children with growth hormone deficiency (GHD), idiopathic short stature (ISS), and Turner Syndrome (TS) who switched from a non-Omnitrope recombinant human growth hormone (rhGH) preparation during routine clinical care.MethodsThis was a retrospective study which identified patients with GHD, ISS, and TS during the study time period of January 1, 2006 and July 31, 2011. Patients were included if they switched to Omnitrope from another non-Omnitrope rhGH therapy during the study time period, were <18 years of age at time of switch, and on a prior rhGH therapy for at least 15 months pre-switch and on Omnitrope for 15 months post-switch. Auxological parameters (height, height standard deviation score [HSDS], height velocity [HV], and height velocity standard deviation score [HVSDS]) were evaluated during post-switch.ResultsOne hundred and three patients were identified: GHD (n = 57), ISS (n = 26), and TS (n = 20). There was continuous growth in height for all 103 patients with an average rate of 6.52 cm over the 15-month post-switch period. Patients with GHD grew an average rate of 6.30 cm, patients with ISS grew an average rate of 6.58 cm, and patients with TS grew an average rate of 6.52 cm over the 15-month post-switch period. The average rate of HSDS was increased by 0.04 for all patients. The HV and HVSDS demonstrated the expected decline with advancing age and prolonged duration of treatment.ConclusionsThe growth trajectories of rhGH-treated patients were not negatively impacted by switching to Omnitrope and growth rates remained as expected prior to the switch.Electronic supplementary materialThe online version of this article (doi:10.1007/s13554-014-0017-1) contains supplementary material, which is available to authorized users.

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Section: Introductionmentioning

confidence: 99%

Switching to Omnitrope® from Other Recombinant Human Growth Hormone Therapies: A Retrospective Study in an Integrated Healthcare System

Rashid

Saenger

et al. 2014

Biol Ther

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“…The results showed that all the ISS children patients having normal GH level (Table 1). In comparison to reduced GH rate in patients with endocrine or skeletal diseases, the normal GH pattern is considered to be characteristic for ISS patients (Dunkel, 2006). Nevertheless, measurement of GH level is not easily applicable to clinical practice.…”

Section: Discussionmentioning

confidence: 99%

Idiopathic Short Stature in Children: A Hospital Based Study

Alharthi¹

2016

J. of Medical Sciences

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“…GH treatment of ISS was approved in 2003, but such use remains controversial; there is a lack of evidence of psychological or physical morbidity or improved well-being with treatment, cost to the society is high, and the pharmacological dosing JOURNAL 66,67 . There is no reason to expect better growth response with rhIGF-I in these patients than with GH, based on the absence of convincing evidence of GH resistance as a cause of their short stature.…”

Section: Other Indications For Igf-i Therapy For Growth Promotionmentioning

confidence: 97%

Insulin-like Growth Factor-I (rhIGF-I) Therapy of Short Stature

Rosenbloom

2008

Journal of Pediatric Endocrinology and Metabolism

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The United States Food and Drug Administration (FDA) approved the use of subcutaneously injected rhIGF-I in late 2005 for treatment of children with severe short stature from growth hormone (GH) insensitivity due to genetic defects in the GH receptor or postreceptor mechanisms or from the development of GH inactivating antibodies. The approval was based on 15 years experience treating these rare conditions with rhIGF-I. Because of the very small numbers of children with these conditions, there has been an effort to justify and promote broader use for rhIGF-I. Attempts to identify GH unresponsiveness in children with idiopathic short stature (ISS) have yielded only a handful of patients with rare genetic disorders. IGF-I treatment for unequivocal GH insensitivity improves but does not correct growth failure, in contrast to the typical experience with GH replacement of GH deficiency. This emphasizes the importance of direct effects of GH at the growth plate, including the stimulation of maturation of cartilage precursor cells and local production of IGF-I, effects that cannot be duplicated by exogenous administration of rhIGF-I. Adverse effects testify to the more than adequate delivery of administered rhIGF-I to other tissues; these include lymphoid hyperplasia, coarsening of the facies, and increased percentage body fat. The absence of convincing evidence of GH insensitivity in a substantial number of children with ISS, the limited ability of endocrine IGF-I to restore normal growth in those with unequivocal GH unresponsiveness, the suppression of endogenous GH (and thereby, local GH effects on growth) that occurs with IGF-I administration, the risk profile, and the absence of data on efficacy in other than proven severe GH insensitivity, led the Drug and Therapeutics Committee of the Lawson Wilkins Pediatric Endocrine Society to conclude that rhIGF-I use is only justified in conditions approved by the FDA and that other growth promotional use should only be investigational. Nonetheless, substantial numbers of children are being treated with rhIGF-I off-label, exuberant estimates of potentially eligible patients are projected, and several uncontrolled clinical trials have been undertaken which are not based on sound preliminary data or established growth principles, and a single four-arm study begun comparing monotherapy with rhGH to combination rhGH with three dosages of rhIGF-I as a single daily injection, a means of administration of rhIGF-I that has not been tested.

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Management of children with idiopathic short stature

Cited by 5 publications

References 26 publications

Switching to Omnitrope® from Other Recombinant Human Growth Hormone Therapies: A Retrospective Study in an Integrated Healthcare System

Switching to Omnitrope® from Other Recombinant Human Growth Hormone Therapies: A Retrospective Study in an Integrated Healthcare System

Idiopathic Short Stature in Children: A Hospital Based Study

Insulin-like Growth Factor-I (rhIGF-I) Therapy of Short Stature

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