The role of recombinant human insulin-like growth factor-I in treating children with short stature

Journal of Clinical Endocrinology and Metabolism

Volumn 93, Issue 1, 2008, Pages 10-18

  Collett Solberg, Paulo F ^a,c   Misra, Madhusmita ^b  

^a Instituto Estadual de Diabetes e Endocrinologia Luiz Capriglione   (Brazil)

^b MASSACHUSETTS GENERAL HOSPITAL   (United States)

^c NONE   (Brazil)

Author keywords

[No Author keywords available]

Indexed keywords

GROWTH HORMONE; RECOMBINANT GROWTH HORMONE; RECOMBINANT SOMATOMEDIN C; SOMATOMEDIN BINDING PROTEIN 3; SOMATOMEDIN C;

ACNE; AMENORRHEA; ANDROGEN BLOOD LEVEL; BODY HEIGHT; CANCER RISK; CLINICAL FEATURE; COMBINATION CHEMOTHERAPY; DISEASE COURSE; DRUG EFFICACY; DRUG INDICATION; DRUG SAFETY; GIGANTISM; GROWTH HORMONE BLOOD LEVEL; GROWTH HORMONE DEFICIENCY; HEADACHE; HUMAN; HYPERANDROGENISM; HYPOGLYCEMIA; INJECTION SITE REACTION; INSULIN RESISTANCE; INTRACRANIAL HYPERTENSION; MONOTHERAPY; OLIGOMENORRHEA; PRIORITY JOURNAL; REVIEW; RISK ASSESSMENT; SHORT STATURE; SIDE EFFECT; TACHYCARDIA; TREATMENT OUTCOME;

EID: 38149136430     PISSN: 0021972X     EISSN: 0021972X     Source Type: Journal    
DOI: 10.1210/jc.2007-1534     Document Type: Review

References (81)

1. Regan F, Kerdanet M, Dattani M, Sommer A, Attie KM, Dunger DB rhIGF-1/rhIGFBP-3 treatment of patients with severe insulin resistance syndromes: preliminary data. Program of the 88th Annual Meeting of The Endocrine Society, Boston, MA, 2006, p 133 (Abstract OR 40-2)
2. Kuzuya H, Matsuura N, Sakamoto M, Makino H, Sakamoto Y, Kadowaki T, Suzuki Y, Kobayashi M, Akazawa Y, Nomura M, et al 1993 Trial of insulin-like growth factor I therapy for patients with extreme insulin resistance syndromes. Diabetes 42:696-705
3. Carroll PV, Christ ER, Umpleby AM, Gowrie I, Jackson N, Bowes SB, Hovorka R, Croos P, Sönksen PH, Russell-Jones DL 2000 IGF-I treatment in adults with type 1 diabetes: effects on glucose and protein metabolism in the fasting state and during a hyperinsulinemic-euglycemic amino acid clamp. Diabetes 49:789-796
4. Acerini CL, Patton CM, Savage MO, Kernell A, Westphal O, Dunger DB 1997 Randomised placebo-controlled trial of human recombinant insulin-like growth factor I plus intensive insulin therapy in adolescents with insulin-dependent diabetes mellitus. Lancet 350:1199-1204
5. Lorenzo-Zuniga V, Rodriguez-Ortigosa CM, Bartoli R, Martinez-Chantar ML, Martinez-Peralta L, Pardo A, Ojanguren I, Quiroga J, Planas R, Prieto J 2006 Insulin-like growth factor I improves intestinal barrier function in cirrhotic rats. Gut 55:1306-1312
6. Conchillo M, de Knegt RJ, Payeras M, Quiroga J, Sangro B, Herrero JI, Castilla-Cortazar I, Frystyk J, Flyvbjerg A, Yoshizawa C, Jansen PL, Scharschmidt B, Prieto J 2005 Insulin-like growth factor I (IGF-I) replacement therapy increases albumin concentration in liver cirrhosis: results of a pilot randomized controlled clinical trial. J Hepatol 43:630-636
7. Saatman KE, Contreras PC, Smith DH, Raghupathi R, McDermott KL, Fernandez SC, Sanderson KL, Voddi M, McIntosh TK 1997 Insulin-like growth factor-1 (IGF-1) improves both neurological motor and cognitive outcome following experimental brain injury. Exp Neurol 147:418-427
8. Carroll PV, Jackson NC, Russell-Jones DL, Treacher DF, Sonksen PH, Umpleby AM 2004 Combined growth hormone/insulin-like growth factor I in addition to glutamine-supplemented TPN results in net protein anabolism in critical illness. Am J Physiol Endocrinol Metab 286:E151-E157
9. Baumann G 1991 Growth hormone heterogeneity: genes, isohormones, variants, and binding proteins. Endocr Rev 12:424-449
10. Lewis UJ, Sinha YN, Haro LS 1994 Variant forms and fragments of human growth hormone in serum. Acta Pediatr 399(Suppl):29-31
11. Nilsson O, Marino R, DeLuca F, Phillip M, Baron J 2005 Endocrine regulation of the growth plate. Horm Res 64:157-165
12. Ohlsson C, Bengtsson B, Isaksson O, Andreassen T, Slootweg M 1998 Growth hormone and bone. Endocr Rev 19:55-79
13. Daughaday WH, Rotwein P 1989 Insulin-like growth factors I and II. Peptide, messenger ribonucleic acid and gene structures, serum, and tissue concentrations. Endocr Rev 10:68-91
14. Cohen P, Rosenfeld RG 1995 The IGF axis. In: Rosenbloom AL, ed. Human growth hormone: basic and scientific aspects. Boca Raton FL: CRC Press; 43-58
15. Collett-Solberg PF, Cohen P 1996 The role of the insulin-like growth factor binding proteins and the IGFBP proteases in modulating IGF action. Endocrinol Metab Clin North Am 25:591-614
16. Baxter RC 1994 Insulin-like growth factor binding proteins in the human circulation: a review. Horm Res 42:140-144
17. Twigg SM, Baxter RC 1998 Insulin-like growth factor (IGF)-binding protein 5 forms an alternative ternary complex with IGFs and the acid-labile subunit. J Biol Chem 273:6074-6079
18. Khosravi MJ, Diamandi A, Mistry J, Krishna RG, Khare A 1997 Acid-labile subunit of human insulin-like growth factor-binding protein complex: measurement, molecular, and clinical evaluation. J Clin Endocrinol Metab 82:3944-3951
19. Hasegawa T, Cohen P, Hasegawa Y, Fielder PJ, Rosenfeld RG 1995 Characterization of the insulin-like growth factors (IGF) axis in a cultured mouse Leydig cell line (TM-3). Growth Regul 5:151-159
20. Arany E, Afford S, Strain AJ, Winwood PJ, Arthur MJ, Hill DJ 1994 Differential cellular synthesis of insulin-like growth factor binding protein-1 (IGFBP-1) and IGFBP-3 within human liver. J Clin Endocrinol Metab 79:1871-1876
21. Chin E, Zhou J, Dai J, Baxter RC, Bondy CA 1994 Cellular localization and regulation of gene expression for components of the insulin-like growth factor ternary binding protein complex. Endocrinology 134:2498-2504
22. Villafuerte BC, Koop BL, Pao CI, Gu L, Birdsong GG, Phillips LS 1994 Coculture of primary rat hepatocytes and nonparenchymal cells permits expression of insulin-like growth factor binding protein-3 in vitro. Endocrinology 134:2044-2050
23. Rosenfeld RG, Pham H, Cohen P, Fielder P, Gargosky SE, Muller H, Nonoshita L, Oh Y 1994 Insulin-like growth factor binding proteins and their regulation. Acta Paediatr Suppl 399:154-158
24. Baxter RC, Binoux MA, Clemmons DR, Conover CA, Drop SL, Holly JM, Mohan S, Oh Y, Rosenfeld RG 1998 Related articles, links recommendations for nomenclature of the insulin-like growth factor binding protein superfamily. Endocrinology 139:4036
25. Cohen P, Fielder PJ, Hasegawa Y, Frisch H, Giudice LC, Rosenfeld RG 1991 Clinical aspects of insulin-like growth factor binding proteins. Acta Endocrinol 124(Suppl 2):74-85
26. Holly JM, Martin JL 1994 Insulin-like growth factor binding proteins: a review of methodological aspects of their purification, analysis and regulation. Growth Regul 4(Suppl 1):20-30
27. Lamson G, Giudice LC, Rosenfeld RG 1991 Insulin-like growth factor binding proteins: structural and molecular relationships. Growth Factors 5:19-28
28. Underwood LE, Thissen JP, Lemozy S, Ketelslegers JM, Clemmons DR 1994 Hormonal and nutritional regulation of IGF-I and its binding proteins. Horm Res 42:145-151
29. Cohen P, Rosenfeld RG 1994 Physiologic and clinical relevance of the insulin-like growth factor binding proteins. Curr Opin Pediatr 6:462-467
30. Savage MO, Woods KA 1996 The investigation of growth hormone insensitivity. Clin Endocrinol (Oxf) 45:257-260
31. Schmid C 1995 Insulin-like growth factors. Cell Biol Int 19:445-457
32. Le Roith D 1997 Seminars in medicine of the Beth Israel Deaconess Medical Center. Insulin-like growth factors. N Engl J Med 336:633-640
33. Lupu F, Terwilliger JD, Lee K, Segre GV, Efstratiadis A 2001 Roles of growth hormone and insulin-like growth factor 1 in mouse postnatal growth. Dev Biol 229:141-162
34. Fielder PJ, Mortensen DL, Mallet P, Carlsson B, Baxter RC, Clark RG 1996 Differential long-term effects of insulin-like growth factor-I (IGF-I) growth hormone (GH), and IGF-I plus GH on body growth and IGF binding proteins in hypophysectomized rats. Endocrinology 137:1913-1920
35. Bright GM, Julius JR, Lima J, Blethen SL 1999 Growth hormone stimulation test results as predictors of recombinant human growth hormone treatment outcomes: preliminary analysis of the national cooperative growth study database. Pediatrics 104:1028-1031
36. Richman RA, Kirsch LR 1988 Testosterone treatment in adolescent boys with constitutional delay in growth and development. N Engl J Med 319:1563-1567
37. Adan L, Souberbielle JC, Brauner R 1994 Management of short stature due to pubertal delay in boys. J Clin Endocrinol Metab 78:478-482
38. Arrigo T, Cisternino M, Luca De F, Saggese G, Messina MF, Pasquino AM, De Sanctis V 1996 Final height outcome in both untreated and testosteronetreated boys with constitutional delay of growth and puberty. J Pediatr Endocrinol Metab 9:511-517
39. Lampit M, Hochberg Z 2003 Androgen therapy in constitutional delay of growth. Horm Res 59:270-275
40. Buckway CK, Guevara-Aguirre J, Pratt KL, Burren CP, Rosenfeld RG 2001 The IGF-I generation test revisited: a marker of GH sensitivity. J Clin Endocrinol Metab 86:5176-5183
41. Blum WF, Cotterill AM, Postel-Vinay MC, Ranke MB, Savage MO, Wilton P 1994 Improvement of diagnostic criteria in growth hormone insensitivity syndrome: solutions and pitfalls. Pharmacia study group on insulin-like growth factor I treatment in growth hormone insensitivity syndromes. Acta Paediatr Suppl 399:117-124
42. Buckway CK, Selva KA, Pratt KL, Tjoeng E, Guevara-Aguirre J, Rosenfeld RG 2002 Insulin-like growth factor binding protein-3 generation as a measure of GH Sensitivity. J Clin Endocrinol Metab 87:4754-4765
43. Selva KA, Buckway CK, Sexton G, Pratt KL, Tjoeng E, Guevara-Aguirre J, Rosenfeld RG 2003 Reproducibility in patterns of IGF generation with special reference to idiopathic short stature. Horm Res 60:237-246
44. Savage MO, Blum WF, Ranke MB, Postel-Vinay MC, Cotterill AM, Hall K, Chatelain PG, Preece MA, Rosenfeld RG 1993 Clinical features and endocrine status in patients with growth hormone insensitivity (Laron syndrome). J Clin Endocrinol Metab 77:1465-1471
45. Rosenfeld RG, Rosenbloom AL, Guevara-Aguirre J 1994 Growth hormone (GH) insensitivity due to primary GH receptor deficiency. Endocr Rev 15:369-390
46. Cotterill AM, Camacho-Hubner C, Duquesnoy P, Savage MO 1998 Changes in serum IGF-I and IGFBP-3 concentrations during the IGF-I generation test performed prospectively in children with short stature. Clin Endocrinol (Oxf) 48:719-724
47. Blair JC, Camacho-Hubner C, Miraki Moud F, Rosberg S, Burren C, Lim S, Clayton PE, Bjarnason R, Albertsson-Wikland K, Savage MO 2004 Standard and low-dose IGF-I generation tests and spontaneous growth hormone secretion in children with idiopathic short stature. Clin Endocrinol (Oxf) 60:163-168
48. Blethen SL, Baptista J, Kuntze J, Foley T, LaFranchi S, Johanson A 1997 Adult height in growth hormone (GH)-deficient children treated with biosynthetic GH. J Clin Endocrinol Metab 82:418-420
49. Reiter EO, Price DA, Wilton P, Albertsson-Wiland K, Ranke MB 2006 Effect of growth hormone (GH) treatment on the near-final height of 1258 patients with idiopathic GH deficiency: analysis of a large international database. J Clin Endocrinol Metab 91:2047-2054
50. Klinger B, Laron Z 1995 Three year IGF-I treatment of children with Laron syndrome. J Ped Endocrinol Metab 8:149-158
51. Backeljauw PF, Underwood LE; GHIS Collaborative Group 2001 Growth hormone insensitivity syndrome. Therapy for 6.5-7.5 years with recombinant insulin-like growth factor I in children with growth hormone insensitivity syndrome: a clinical research center study. J Clin Endocrinol Metab 86:1504-1510
52. Vaccarello MA, Diamond Jr FB, Guevara-Aguirre J, Rosenbloom AL, Fielder PJ, Gargosky S, Cohen P, Wilson K, Rosenfeld RG 1993 Hormonal and metabolic effects and pharmacokinetics of recombinant insulin-like growth factor-I in growth hormone receptor deficiency/Laron syndrome. J Clin Endocrinol Metab 77:273-280
53. Domené HM, Bengolea SV, Martínez AS, Ropelato MG, Pennisi P, Scaglia P, Heinrich JJ, Jasper HG 2004 Brief report: deficiency of the circulating insulin-like growth factor system associated with inactivation of the acid-labile subunit gene. N Engl J Med 350:570-577
54. Domené HM, Martínez AS, Frystyk J, Bengolea SV, Ropelato MG, Scaglia PA, Chen JW, Heuck C, Wolthers OD, Heinrich JJ, Jasper HG 2007 Normal growth spurt and final height despite low levels of all forms of circulating insulin-like growth factor-I in a patient with acid-labile subunit deficiency. Horm Res 67:243-249
55. Ranke MB, Savage MO, Chatelain PG, Preece MA, Rosenfeld RG, Wilton P 1999 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 51:128-134
56. Savage MO, Attie KM, David A, Metherell LA, Clark AJ, Camacho-Hubner C 2006 Endocrine assessment, molecular characterization and treatment of growth hormone insensitivity disorders. Nat Clin Pract Endocrinol Metab 2:395-407
57. Chernausek SD, Backeljauw PF, Frane J, Kuntze J, Underwood LE 2007 Long-term treatment with recombinant IGF-I in children with severe IGF-I deficiency due to growth hormone insensitivity. J Clin Endocrinol Metab 92:902-910
58. Guevara-Aguirre J, Rosenbloom AL, Vasconez O, Martinez V, Gargosky SE, Allen L, Rosenfeld RG 1997 Two-year treatment of growth hormone (GH) receptor deficiency 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 82:629-633
59. Riedl S, Frisch H 2006 Effects of growth hormone (GH) and insulin-like growth factor-I therapy in patients with gene defects in the GH axis. J Pediatr Endocrinol Metab 19:229-236
60. Camacho-Hubner C, Underwood LE, Yordam N, Yuksel B, Smith AV, Attie KM, Savage MO Once daily rhIGF-I/rhIGFBP-3 treatment improves growth velocity in children with severe primary IGF-I deficiency: results of a multicenter clinical trial. Program of the 88th Annual Meeting of The Endocrine Society, Boston, MA, 2006 (Abstract OR 40-1)
61. Woods KA, Camacho-Hubner C, Bergman RN, Barter D, Clark AJ, Savage MO 2000 Effects of insulin-like growth factor I (IGF-I) therapy on body composition and insulin resistance in IGF-I gene deletion. J Clin Endocrinol Metab 85:1407-1411
62. Blethen S, Hertz J, Kuntze J, Rotenstein D, Serrat J The Increlex Growth Forum Database Registry: a Web-based patient registry for monitoring long-term safety and efficacy of Increlex treatment. Program of the 89th Annual Meeting of The Endocrine Society, Toronto, Canada, 2007, p 275 (Abstract P1-466)
63. Attie KM, Carlsson LM, Rundle AC, Sherman BM 1995 Evidence for partial growth hormone insensitivity among patients with idiopathic short stature. The National Cooperative Growth Study. J Pediatr 127:244-250
64. Goddard AD, Dowd P, Chernausek S, Geffner M, Gertner J, Hintz R, Hopwood N, Kaplan S, Plotnick L, Rogol A, Rosenfield R, Saenger P, Mauras N, Hershkopf R, Angulo M, Attie K 1997 Partial growth-hormone insensitivity: the role of growth-hormone receptor mutations in idiopathic short stature. J Pediatr 131:S51-S55
65. Cohen P, Bright GM, Rogol AD, Kappelgaard AM, Rosenfeld RG; American Norditropin Clinical Trials Group 2002 Effects of dose and gender on the growth and growth factor response to GH in GH-deficient children: implications for efficacy and safety. J Clin Endocrinol Metab 87:90-98
66. Cohen P, Rogol AD, Howard CP, Bright GM, Kappelgaard AM, Rosenfeld RG 2007 IGF-based dosing of GH therapy in children: a randomized controlled study. J Clin Endocrinol Metab 92:2480-2486
67. Ranke MB, Lindberg A, Price DA, Darendeliler F, Albertsson-Wikland K, Wilton P, Reiter EO 2007 KIGS International Board. Age at growth hormone therapy start and first-year responsiveness to growth hormone are major determinants of height outcome in idiopathic short stature. Horm Res 68:53-62
68. Kristrom B, Lofqvist C, Rosberg S, Albertsson Wikland K 2001 Effect of spontaneous GH secretion and the GH sampling period on the accuracy of models for predicting growth responses to GH treatment. J Clin Endocrinol Metab 86:4963-4964
69. Simpson HL, Jackson NC, Shojaee-Moradie F, Jones RH, Russell-Jones DL, Sonksen PH, Dunger DB, Umpleby AM 2004 Insulin-like growth factor I has a direct effect on glucose and protein metabolism, but no effect on lipid metabolism in type 1 diabetes. J Clin Endocrinol Metab 89:425-432
70. Møller N, Blum WF, Mengel A, Hansen LB, Alberti KGMM, Schmitz O 1991 Basal and insulin stimulated substrate metabolism in tumour induced hypoglycaemia; evidence for increased muscle glucose uptake. Diabetologia 34:17-20
71. Guler HP, Zapf J, Froesch ER 1987 Short-term metabolic effects of recombinant human insulin-like growth factor I in healthy adults. N Engl J Med 317:137-140
72. Guevara-Aguirre J, Vasconez O, Martinez V, Martinez AL, Rosenbloom AL, Diamond Jr FB, Gargosky SE, Nonoshita L, Rosenfeld RG 1995 A randomized double-blind, placebo-controlled trial of safety and efficacy of recombinant insulin-like growth factor-I in children with growth hormone receptor deficiency. J Clin Endocrinol Metab 80:1393-1398
73. Guevara-Aguirre J, Guevara-Aguirre M, Rosenbloom AL 2006 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 Pædiatr 95:199-202
74. Vasconez O, Martinez V, Martinez AL, Hidalgo F, Diamond FB, Rosenbloom AL, Rosenfeld RG, Guevara-Aguirre J 1994 Heart rate increase in patients with growth hormone receptor deficiency treated with insulin-like growth factor I. Acta Paediatr Suppl 399:137-139
75. Laron Z, Ginsberg S, Lilos P, Arbiv M, Vaisman N 2006 Long-term IGF-I treatment of children with Laron syndrome increases adiposity. Growth Horm IGF Res 16:61-64
76. Klinger B, Anin S, Silbergeld A, Eshet R, Laron Z 1998 Development of hyperandrogenism during treatment with insulin-like growth factor-I (IGF-I) in female patients with Laron syndrome. Clin Endocrinol (Oxf) 48:81-87
77. Perry JK, Emerald BS, Mertani HC, Lobie PE 2006 The oncogenic potential of growth hormone. Growth Horm IGF Res 16:277-289
78. Samani AA, Yakar S, LeRoith D, Brodt P 2007 The role of the IGF system in cancer growth and metastasis: overview and recent insights. Endocr Rev 28:20-47
79. Rogol AD, Midyett LK, Van Meter Q, Frane J, Bailly J, Bright GM Recombinant human IGF-I for children with primary IGF-I deficiency: safety data from ongoing clinical trials. Program of the Pediatric Academic Societies Annual Meeting, Toronto, 2007 (Publication 6305.13)
80. Rosenbloom AL, Guevara-Aguirre J 2006 Controversy in clinical endocrinology: reclassification of insulin-like growth factor I production and action disorders. J Clin Endocrinol Metab 91:4232-4234
81. Cohen P 2006 Controversy in clinical endocrinology: problems with reclassification of insulin-like growth factor I production and action disorders. J Clin Endocrinol Metab 91:4235-4236