Recombinant Insulin-Like Growth Factor (IGF)-I Treatment in Short Children with Low IGF-I Levels: First-Year Results from a Randomized Clinical Trial

Midyett, L. Kurt; Rogol, Alan D.; Meter, Quentin L. Van; Frane, James W.; Bright, George M.

doi:10.1210/jc.2009-0570

Cited by 64 publications

(64 citation statements)

References 28 publications

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“…at the upper limit of international recommendations (39); accelerated bone maturation compared with the control group (1.1 and 1.2 vs 0.8 years), and a significantly greater number of children entering puberty (12 vs 1). However, this study (8) demonstrates the efficacy of rhIGF1, with a growth rate increase in both dosage groups (7 and 7.9 vs 5.2 cm/year in controls) and increased height (C0. 4 The prevalence of severe primary IGFD defined using criteria for rhIGF1 treatment was only 1.2% in a vast cohort of children with short stature.…”

Section: European Journal Of Endocrinologymentioning

confidence: 52%

“…Growth rates increased by 5.4-6.1 cm/year according to the dosage (80 mg/kg-120 mg/kg per 12 h) (38). A single study evaluated the efficacy of rhIGF1 given for 1 year to children with ISS (8). These results indicate a need for caution regarding several points such as the mean IGF1 SDS increases of C2 (80 mg/kg) or C2.2 (120 mg/kg), i.e.…”

Section: European Journal Of Endocrinologymentioning

confidence: 99%

“…Treatment with rhIGF1 was first used in children with severe primary IGFD caused by insensitivity to GH (Laron syndrome, IGF1 gene mutations, or GH-neutralizing antibodies) (6,7,8). Add-on rhIGF1 therapy improved the growth rate and final height of these patients who were not eligible for GH therapy.…”

Section: Introductionmentioning

confidence: 99%

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Characterization and prevalence of severe primary IGF1 deficiency in a large cohort of French children with short stature

Teissier

Flechtner

Colmenares

et al. 2014

European Journal of Endocrinology

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Objective: The prevalence of severe primary IGF1 deficiency (IGFD) is unclear. IGFD must be identified promptly as treatment with recombinant human IGF1 (rhIGF1) is now available. Our objective was to characterize and assess the prevalence of severe primary IGFD in a large cohort of patients evaluated for short stature at a pediatric endocrinology unit in France. Design: Observational study in a prospective cohort. Methods: Consecutive patients referred to our unit between 2004 and 2009 for suspected slow statural growth were included. Patients were classified into eight etiological categories. IGFD was defined by height %K3 SDS, serum IGF1 levels !2.5th percentile, GH sufficiency, and absence of causes of secondary IGFD. Results: Out of 2546 patients included, 337 (13.5%) were born small for gestational age and 424 (16.9%) had idiopathic short stature. In these two categories, we identified 30 patients who met our criterion for IGFD (30/2546, 1.2%). In these 30 patients, we assessed the response to IGF1 generation test, time course of IGF1 levels, and efficiency of GH replacement therapy. The results indicated that only four of the 30 children were definite or possible candidates for rhIGF1 replacement therapy. Conclusion: The prevalence of severe primary IGFD defined using the standard criterion for rhIGF1 treatment was 1.2%, and only 0.2% of patients were eligible for rhIGF1 therapy.

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Section: European Journal Of Endocrinologymentioning

confidence: 52%

Section: European Journal Of Endocrinologymentioning

confidence: 99%

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Characterization and prevalence of severe primary IGF1 deficiency in a large cohort of French children with short stature

Teissier

Flechtner

Colmenares

et al. 2014

European Journal of Endocrinology

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“…73 There was no rhGH-treatment comparison group in that study, although it has been suggested that rhGH treatment of children with ISS may be expected to produce a more positive effect on adult height as it does not lead to advancement of osseous maturation compared with controls. The mean increments in growth velocities attained in this study (1.8cm/year and 2.7cm/year for the two treatment groups) have also been noted to be lower than the growth velocities achieved in patients with severe IGF-1 deficiency due to GH insensitivity treated with rhIGF-1 at the same doses (5.4 and 6.1cm/year, respectively).…”

Section: Recombinant Human Insulin-like Growth Factor-i Height Attainmentioning

confidence: 90%

Specialty Drugs for the Treatment of Short Stature in Children—Pros, Cons, and Perspectives for the Future

Koontz¹,

Cuttler

2010

US Endocrinology

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Specialty drugs are generally defined as medications that involve special drug handling and/or parenteral administration and are typically used to treat complex medical conditions. They are typically biologicals, often very expensive, and generally prescribed by specialists. The recent surge in use of specialty pharmaceuticals has placed these drugs in the spotlight as policy-makers struggle to contain healthcare costs. Specialty drugs are central to discussions about optimal ways to manage childhood short stature; recombinant human growth hormone (rhGH) and recombinant human insulin-like growth factor-1 (rhIGF-1)—specialty drugs with annual prices of $20,000 to $30,000 per child—are available to treat childhood short stature from specific causes. rhGH and rhIGF-1 revolutionized treatment of severe short stature resulting from growth hormone deficiency and growth hormone insensitivity, respectively. Over the past 20 years, use of rhGH has expanded to other conditions. Expanded use of the newer rhIGF-1 may occur in an analogous manner. This article reviews the background, current status, and potential for these drugs in view of current evidence and policies.

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“…The reports of IH in children with severe GH insensitivity (Laron syndrome) and children with low insulin-like growth factor 1 (IGF-1) treated with recombinant IGF-1 (19,20) suggest that exposure to IGF-1 may be the pathophysiologic mediator of the rhGH effect. Increased cerebrospinal fl uid (CSF) production, decreased CSF absorption, changes in CSF fl ow, or a combination of these factors have been postulated (21,22) .…”

Section: Discussionmentioning

confidence: 99%

Intracranial hypertension in pediatric patients treated with recombinant human growth hormone: data from 25 years of the Genentech National Cooperative Growth Study

Noto¹,

Maneatis²,

Frane³

et al. 2011

Journal of Pediatric Endocrinology and Metabolism

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Intracranial hypertension (IH) is a rare condition in children. However, a relationship between recombinant human growth hormone (rhGH) therapy and IH has been well documented. Risk factors were assessed for 70 rhGH-naive patients enrolled in the National Cooperative Growth Study with reports of IH after treatment initiation. Patients with severe growth hormone defi ciency, Turner syndrome, chronic renal insuffi ciency (CRI), and obesity (particularly in the CRI group) were at highest risk of developing IH during the fi rst year of therapy, suggesting initiation of careful early monitoring. In some patients, factors such as corticosteroid use or other chromosomal abnormalities appear to confer a delayed risk of IH, and these patients should be monitored long-term for signs and symptoms of IH.

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Recombinant Insulin-Like Growth Factor (IGF)-I Treatment in Short Children with Low IGF-I Levels: First-Year Results from a Randomized Clinical Trial

Abstract: rhIGF-I treatment was associated with age- and dose-dependent increases in first-year height velocity. Adverse events during treatment were less common than in previous studies and were generally transient, easily managed, and without known sequelae.

Cited by 64 publications

References 28 publications

Characterization and prevalence of severe primary IGF1 deficiency in a large cohort of French children with short stature

Characterization and prevalence of severe primary IGF1 deficiency in a large cohort of French children with short stature

Specialty Drugs for the Treatment of Short Stature in Children—Pros, Cons, and Perspectives for the Future

Intracranial hypertension in pediatric patients treated with recombinant human growth hormone: data from 25 years of the Genentech National Cooperative Growth Study

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