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33746867985
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Rosenbloom AL. Is there a role for recombinant insulin-like growth factor-I (rhIGF-I) in the treatment of idiopathic short stature? Lancet 2006; 368:612-616. This viewpoint article extensively quotes company reports to the US Securities and Exchange Commission and market analyst commentary to indicate the promotional exuberance beyond the approved indications and discusses problems with the term 'primary IGFD', lack of evidence for widespread application of the term and concerns about treatment of ISS with rhIGF-I.
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Rosenbloom AL. Is there a role for recombinant insulin-like growth factor-I (rhIGF-I) in the treatment of idiopathic short stature? Lancet 2006; 368:612-616. This viewpoint article extensively quotes company reports to the US Securities and Exchange Commission and market analyst commentary to indicate the promotional exuberance beyond the approved indications and discusses problems with the term 'primary IGFD', lack of evidence for widespread application of the term and concerns about treatment of ISS with rhIGF-I.
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Rosenbloom AL. Recombinant human insulin-like growth factor-I (rhIGF-I) and rhIGF-I/rhIGF-I-binding-protein-3: new growth treatment options? J Pediatr 2007; 150:7-11. This medical progress article reviews the role of IGF-I in growth, the causes and diagnosis of IGF-I deficiency, the experience of treatment with rhIGF-I, including adverse events, and discusses the role of the two available preparations in growth therapy.
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Rosenbloom AL. Recombinant human insulin-like growth factor-I (rhIGF-I) and rhIGF-I/rhIGF-I-binding-protein-3: new growth treatment options? J Pediatr 2007; 150:7-11. This medical progress article reviews the role of IGF-I in growth, the causes and diagnosis of IGF-I deficiency, the experience of treatment with rhIGF-I, including adverse events, and discusses the role of the two available preparations in growth therapy.
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33745190042
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Endocrinology-the way we were: A personal history of somatomedin
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A venerable founding father of the field provides a concise history of the identification of IGF-I and the development and amplification of the somatomedin hypothesis
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Daughaday WH. Endocrinology-the way we were: a personal history of somatomedin. Growth Horm IGF Res 2006; 16:S3-S5. A venerable founding father of the field provides a concise history of the identification of IGF-I and the development and amplification of the somatomedin hypothesis.
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Growth Horm IGF Res
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Daughaday, W.H.1
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van der Eerden, Karperien M, Wit JM. Systemic and local regulation of the growth plate. Endocr Rev 2003; 24:782-801.
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van der Eerden, Karperien M, Wit JM. Systemic and local regulation of the growth plate. Endocr Rev 2003; 24:782-801.
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Rosenbloom AL, Guevara-Aguirre J. Controversy in clinical endocrinology: reclassification of IGF-1 production and action disorders. J Clin Endocrinol Metab 2006; 91:4232-4234. These authors present arguments for avoiding the ambiguous and imprecise terms primary and secondary by simply specifying the abnormalities, as is already the system for much of endocrinology and for the abnormalities affecting GH secretion.
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Rosenbloom AL, Guevara-Aguirre J. Controversy in clinical endocrinology: reclassification of IGF-1 production and action disorders. J Clin Endocrinol Metab 2006; 91:4232-4234. These authors present arguments for avoiding the ambiguous and imprecise terms primary and secondary by simply specifying the abnormalities, as is already the system for much of endocrinology and for the abnormalities affecting GH secretion.
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Controversy in clinical endocrinology: Problems with reclassification of insulin-like growth factor-I production and action disorders
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The author proposes that reclassification is inappropriate until further delineation of growth disorders is available and then only on the basis of consensus from relevant organizations
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Cohen P. Controversy in clinical endocrinology: problems with reclassification of insulin-like growth factor-I production and action disorders. J Clin Endocrinol Metab 2006; 91:4235-4236. The author proposes that reclassification is inappropriate until further delineation of growth disorders is available and then only on the basis of consensus from relevant organizations.
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New molecular mechanisms of GH resistance
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Rosenfeld RG, Hwa V. New molecular mechanisms of GH resistance. Eur J Endocrinol 2004; 151 (suppl):S11-S15.
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Rosenfeld RG. The IGF system: new developments relevant to pediatric practice. Endoc Dev 2005; 9:1-10.
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Hwa V, Haeusler G, Pratt KL, et al. Total absence of functional acid labile subunit, resulting in severe insulin like growth factor deficiency and moderate growth failure. J Clin Endocrinol Metab 2006; 91:1826-1831. This is the second reported case of ALS mutation with severe IGF-I and IGFBP3 deficiency and little, if any, effect on growth. It is difficult to consider this moderate growth failure when the boy's height SDS was 0.4 greater than the mean parental SDS.
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Hwa V, Haeusler G, Pratt KL, et al. Total absence of functional acid labile subunit, resulting in severe insulin like growth factor deficiency and moderate growth failure. J Clin Endocrinol Metab 2006; 91:1826-1831. This is the second reported case of ALS mutation with severe IGF-I and IGFBP3 deficiency and little, if any, effect on growth. It is difficult to consider this moderate growth failure when the boy's height SDS was 0.4 greater than the mean parental SDS.
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14644394915
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Recombinant insulin-like growth factor-I as a therapy for IGF-I deficiency in renal failure
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Clark RG. Recombinant insulin-like growth factor-I as a therapy for IGF-I deficiency in renal failure. Pediatr Nephrol 2005; 20:290-294.
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Clark, R.G.1
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33745918618
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Endocrine assessment, molecular characterization and treatment of growth hormone insensitivity disorders
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This is a comprehensive, readable, and up-to-date review of mutations affecting GH sensitivity, treatment experience with rhIGF-I and considerations that its use in ISS is 'a matter of current debate
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Savage MO, Attie KM, David A, et al. Endocrine assessment, molecular characterization and treatment of growth hormone insensitivity disorders. Nat Clin Practice Endocrinol Metab 2006; 2:395-407. This is a comprehensive, readable, and up-to-date review of mutations affecting GH sensitivity, treatment experience with rhIGF-I and considerations that its use in ISS is 'a matter of current debate.'
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Nat Clin Practice Endocrinol Metab
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Savage, M.O.1
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Defining insulin-like growth factor-I deficiency
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Ranke MB. Defining insulin-like growth factor-I deficiency. Horm Res 2006; 65 (suppl 1):9-14.
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Variability and reliability of single serum IGF-I measurements: Impact on determining predictability of risk ratios in disease management
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Milani D, Carmichael JD, Welkowitz J, et al. Variability and reliability of single serum IGF-I measurements: impact on determining predictability of risk ratios in disease management. J Clin Endocrinol Metab 2004; 89:2271-2274.
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Milani, D.1
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Pitfalls of immunoassay and sample for IGF-I: Comparison of different assay methodologies using various fresh and stored serum samples
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Khosravi J, Diamandi A, Bodani U, et al. Pitfalls of immunoassay and sample for IGF-I: comparison of different assay methodologies using various fresh and stored serum samples. Clin Biochem 2005; 38:659-666.
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Evidence for hypermetabolism in boys with constitutional delay of growth and maturation
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This is a comprehensive analysis demonstrating increased overall energy expenditure in boys with constitutional delay of growth and maturation. They also had lower IGF-I and testosterone than age-matched controls but were comparable with height-matched controls
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Han JC, Balagopal P, Sweeten S, et al. Evidence for hypermetabolism in boys with constitutional delay of growth and maturation. J Clin Endocrinol Metab 2006; 91:2081-2086. This is a comprehensive analysis demonstrating increased overall energy expenditure in boys with constitutional delay of growth and maturation. They also had lower IGF-I and testosterone than age-matched controls but were comparable with height-matched controls.
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J Clin Endocrinol Metab
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Long-term treatment of growth hormone insensitivity syndrome with IGF-I. Results of the European Multicentre Study. The Working Group on Growth Hormone Insensitivity Syndromes
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Ranke MB, Savage MO, Chatelain PG, et al. Long-term treatment of growth hormone insensitivity syndrome with IGF-I. Results of the European Multicentre Study. The Working Group on Growth Hormone Insensitivity Syndromes. Horm Res 1999; 51:128-134.
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Guevara-Aguirre J, Rosenbloom AL, Vasconez O, et al. Two year treatment of GH receptor deficiency (GHRD) with recombinant insulin-like growth factor-I in 22 children: comparison of two dosage levels and to GH treated GH deficiency. J Clin Endocrinol Metab 1997; 82:629-633.
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Chernausek SD, Backeljauw PF, Frane J, et al. Long-term treatment with recombinant IGF-I in children with severe IGF-I deficiency due to growth hormone insensitivity. J Clin Endocrin Metab 2007; 92:902-910. This is the largest series of patients treated with rhIGF-I for the approved indications, providing 1-year data for 59 subjects and as long as 8 years for 14 of them. Six of the patients treated for 5.5-10 years who were at near-final height had height SDS improvements of 0.8-4.3, with the greatest improvement being in the individuals with the lowest initial height SDS. Only two of the individuals had a near-final height SDS above -4.8.
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Chernausek SD, Backeljauw PF, Frane J, et al. Long-term treatment with recombinant IGF-I in children with severe IGF-I deficiency due to growth hormone insensitivity. J Clin Endocrin Metab 2007; 92:902-910. This is the largest series of patients treated with rhIGF-I for the approved indications, providing 1-year data for 59 subjects and as long as 8 years for 14 of them. Six of the patients treated for 5.5-10 years who were at near-final height had height SDS improvements of 0.8-4.3, with the greatest improvement being in the individuals with the lowest initial height SDS. Only two of the individuals had a near-final height SDS above -4.8.
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Camacho-Hübner C, Underwood LE, Yordam N, et al. Once daily rhIGF-I/rhIGFBP 3 treatment improves growth in children with severe primary IGF-I deficiency: results of a multicenter clinical trial. Presented at The Endocrine Society, 27 June 2006; Boston, Massachusetts. http://www. abstracts2view.com/Endo/view.php?nu=ENDO06L_OR40-1 This is the first 1-year treatment report for combination therapy in 25 subjects, for which only 6-month data were available at the time of FDA approval.
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Camacho-Hübner C, Underwood LE, Yordam N, et al. Once daily rhIGF-I/rhIGFBP 3 treatment improves growth in children with severe primary IGF-I deficiency: results of a multicenter clinical trial. Presented at The Endocrine Society, 27 June 2006; Boston, Massachusetts. http://www. abstracts2view.com/Endo/view.php?nu=ENDO06L_OR40-1 This is the first 1-year treatment report for combination therapy in 25 subjects, for which only 6-month data were available at the time of FDA approval.
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Camacho-Hübner C, Rose S, Preece MA, et al. Pharmacokinetic studies of recombinant human insulin-like growth factor I (rhIGF-I)/rhIGF- binding protein-3 complex administered to patients with growth hormone insensitivity syndrome. J Clin Endocrinol Metab 2006; 91:1246-1253. Not only does this study demonstrate the feasibility of a single daily injection of the equimolar combination of rhIGF-I and rhIGFBP3, it also demonstrates the favorable pharmacokinetics of the twice-daily injection of rhIGF-I alone in the same GHRD patients.
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Camacho-Hübner C, Rose S, Preece MA, et al. Pharmacokinetic studies of recombinant human insulin-like growth factor I (rhIGF-I)/rhIGF- binding protein-3 complex administered to patients with growth hormone insensitivity syndrome. J Clin Endocrinol Metab 2006; 91:1246-1253. Not only does this study demonstrate the feasibility of a single daily injection of the equimolar combination of rhIGF-I and rhIGFBP3, it also demonstrates the favorable pharmacokinetics of the twice-daily injection of rhIGF-I alone in the same GHRD patients.
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Pharmacokinetic studies of rhIGF-I/rhIGFBP-3 complex administered to patients with growth hormone insensitivity syndrome (GHIS)
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85th Annual Meeting of the Endocrine Society; Philadelphia, Pennsylvania; June 2003. Chevy Chase
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Guevara-Aguirre J, Guevara-Aguirre M, Rosenbloom AL. Absence of hypoglycemia in response to varying doses of recombinant human insulin-like growth factor-I (rhIGF-I) in children and adolescents with low serum concentrations of IGF-I. Acta Pædiatrica 2006; 95:199-200. Normal adolescents (n=18) with serum IGF-I concentrations of less than -2 SD for age and sex and IGFBP3 below the 50th percentile had no clinical hypoglycemia during 2 weeks of rhIGF-I administration at therapeutic doses, nor during 24-h monitoring at the end of the treatment period.
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Guevara-Aguirre J, Guevara-Aguirre M, Rosenbloom AL. Absence of hypoglycemia in response to varying doses of recombinant human insulin-like growth factor-I (rhIGF-I) in children and adolescents with low serum concentrations of IGF-I. Acta Pædiatrica 2006; 95:199-200. Normal adolescents (n=18) with serum IGF-I concentrations of less than -2 SD for age and sex and IGFBP3 below the 50th percentile had no clinical hypoglycemia during 2 weeks of rhIGF-I administration at therapeutic doses, nor during 24-h monitoring at the end of the treatment period.
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Laron Z, Ginsberg S, Lilos P, et al. Long-term IGF-I treatment of children with Laron syndrome increases adiposity. Growth Horm IGF Res 2006; 16:61-64. This is the first demonstration by dual-energy X-ray absorptiometry of what had previously been implied by following ideal body weight for height, that IGF-I is lipogenic, in contrast to the lipolytic effect of GH in GHD.
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Laron Z, Ginsberg S, Lilos P, et al. Long-term IGF-I treatment of children with Laron syndrome increases adiposity. Growth Horm IGF Res 2006; 16:61-64. This is the first demonstration by dual-energy X-ray absorptiometry of what had previously been implied by following ideal body weight for height, that IGF-I is lipogenic, in contrast to the lipolytic effect of GH in GHD.
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The role of IGF systemin cancer growth and metastasis: Overview and recent insights
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Endocr Rev, this is a comprehensive review of a highly complex subject, noting sufficient epidemiologic and experimental information about mitogenic potential of elevated IGF-I levels to suggest caution about enthusiastic therapeutic provision of IGF-I beyond absolute indications
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Samani AA, Yakar S, LeRoith D, Brodt P. The role of IGF systemin cancer growth and metastasis: overview and recent insights. Endocr Rev 2007; 28:20-47. As would be expected for an Endocrine Reviews article, this is a comprehensive review of a highly complex subject, noting sufficient epidemiologic and experimental information about mitogenic potential of elevated IGF-I levels to suggest caution about enthusiastic therapeutic provision of IGF-I beyond absolute indications.
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As would be expected for an Endocrine Reviews article
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Samani, A.A.1
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A reviewof the growing body of recent literature indicating what the authors describe as a sinister role for GH in oncogenic transformation and neoplastic progression
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Perry JK, Emerald BS, Mertani HC, Lovie PE. The oncogenic potential of growth hormone. Growth Horm IGF Res 2006; 16:277-289. A reviewof the growing body of recent literature indicating what the authors describe as a sinister role for GH in oncogenic transformation and neoplastic progression.
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Park P, Cohen P. Insulin-like growth factor I (IGF-I) measurements in growth hormone (GH) therapy of idiopathic short stature (ISS). Growth Horm IGF Res 2005; 15:S13-S32.
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Limal J-M, Parfait B, Cabrol S, et al. Noonan syndrome: relationships between genotype, growth, and growth factors. J Clin Endocrinol Metab 2006; 91:300-306. Among 35 patients with Noonan syndrome, 20 had PTPN11 missense mutations and, compared with the other patients with Noonan syndrome, had lower birth length, shorter stature, and decreased response to rhGH. IGF-I and ALS levels were low, with normal IGFBP3, suggesting GH resistance due to a late post-receptor signaling defect.
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Limal J-M, Parfait B, Cabrol S, et al. Noonan syndrome: relationships between genotype, growth, and growth factors. J Clin Endocrinol Metab 2006; 91:300-306. Among 35 patients with Noonan syndrome, 20 had PTPN11 missense mutations and, compared with the other patients with Noonan syndrome, had lower birth length, shorter stature, and decreased response to rhGH. IGF-I and ALS levels were low, with normal IGFBP3, suggesting GH resistance due to a late post-receptor signaling defect.
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Hujeirat Y, Hess O, Shalev S, Tenenbaum-Rakover Y. Growth hormone receptor sequence changes do not play a role in determining height in children with idiopathic short stature. Horm Res 2006; 65:210-216. Thirty-three out of 150 children with ISS who had biochemical characteristics suggestive of GH insensitivity were studied and two were found to have polymorphisms that did not affect the height of other family members.
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Guevara-Aguirre J, Rosenbloom AL, Guevara-Aguirre M, et al. Effects of heterozygosity for the E180 splice mutation causing growth hormone receptor deficiency in Ecuador on IGF-I, IGFBP-3, and stature. Growth Horm IGF Res 2007 (in press). IGF-I and IGFBP3 synthesis was not affected by heterozygosity for the E180 splice mutation that causes GHRD/Laron syndrome in the Ecuadorian population. Heterozygosity was associated with reduction in mean statural SDS, but this effect was not sufficient to be clinically important and not mediated through measurable differences in circulating IGF-I or IGFBP3 related to genotype.
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