Growth Hormone: The Expansion of Available Products and Indications

Pediatric Clinics of North America

Volumn 58, Issue 5, 2011, Pages 1141-1165

  Franklin, Sherry L ^a   Geffner, Mitchell E ^b  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

Author keywords

Growth hormone; Indications; ISS; Prader Willi syndrome; SHOX; Small for gestational age; Turner syndrome

Indexed keywords

ANSOMONE; FITROPIN; GROWTH HORMONE; HUMAN GROWTH HORMONE; HYPERTROPIN; SOMATREM; TEVTROPIN; UNCLASSIFIED DRUG;

AMINO ACID SEQUENCE; CHRONIC KIDNEY FAILURE; DNA RECOMBINATION; DRUG APPROVAL; DRUG INDICATION; GROWTH HORMONE DEFICIENCY; HUMAN; IDIOPATHIC SHORT STATURE; MUSCLE ATROPHY; NOONAN SYNDROME; PRADER WILLI SYNDROME; RECOMMENDED DRUG DOSE; REVIEW; SHORT BOWEL SYNDROME; SHORT STATURE; SHORT STATURE HOMEOBOX CONTAINING GENE DEFICIENCY; SMALL FOR DATE INFANT; TURNER SYNDROME; UNITED STATES;

EID: 80053602372     PISSN: 00313955     EISSN: 15578240     Source Type: Journal    
DOI: 10.1016/j.pcl.2011.07.006     Document Type: Review

References (92)

1. McKusick V.A., Rimoin D.L. General Thom Thumb and other midgits. Sci Am 1967, 217:102-106.
2. Raben M.S. Treatment of a pituitary dwarf with human growth hormone. J Clin Endocrinol Metab 1958, 18:901-903.
3. Frasier S.D. The not-so-good old days: working with pituitary growth hormone in North America, 1956 to 1985. J Pediatr 1997, 131:S1-S4.
4. Report of the Committee on Growth Hormone Use of the Lawson Wilkins Pediatric Endocrine Society. Degenerative neurologic disease in patients formerly treated with human growth hormone. J Pediatr 1985, 107:10-12.
5. Flodh H. Human growth hormone produced with recombinant DNA technology: development and production. Acta Paediatr Scand Suppl 1986, 325:1-9.
6. Cronin M.J. Pioneering recombinant growth hormone manufacturing: pounds produced per mile of height. J Pediatr 1997, 131:S5-S7.
7. Massa G., Vanderschueren-Lodeweyckx M., Bouillon R. Five-year follow-up of growth hormone antibodies in growth hormone-deficient children treated with recombinant human growth hormone. Clin Endocrinol 1993, 38:137-142.
8. Takahashi Y., Kaji H., Okimura Y., et al. Short stature caused by a mutant growth hormone. N Engl J Med 1996, 334:432-436.
9. Dattani M.T. Novel insights into the aetiology and pathogenesis of hypopituitarism. Horm Res 2004, 62(Suppl 3):1-13.
10. Traggiai C., Stanhope R. Endocrinopathies associated with midline cerebral and cranial malformations. J Pediatr 2002, 140:252-255.
11. Costin G. Endocrine disorders associated with tumors of the pituitary and hypothalamus. Pediatr Clin North Am 1979, 26:15-31.
12. Mayfield R.K., Levine J.H., Gordon L., et al. Lymphoid adenohypophysitis presenting as a pituitary tumor. Am J Med 1980, 69:619-623.
13. Bartsocas C.S., Pantelakis S.N. Human growth hormone therapy in hypopituitarism due to tuberculous meningitis. Acta Paediatr Scand 1973, 62:304-306.
14. Stuart C.A., Neelon F.A., Lebovitz H.E. Hypothalamic insufficiency: the cause of hypopituitarism in sarcoidosis. Ann Intern Med 1978, 88:589-594.
15. Donadieu J., Rolon M.A., Pion I., French LCH Study Group, et al. Incidence of growth hormone deficiency in pediatric-onset Langerhans cell histiocytosis: efficacy and safety of growth hormone treatment. J Clin Endocrinol Metab 2004, 89:604-609.
16. Lewis A.S., Courtney C.H., Atkinson A.B. All patients with idiopathic hypopituitarism should be screened for hemochromatosis. Pituitary 2008, 12:273-275.
17. Aimaretti G., Ambrosio M.R., Benvenga S., et al. Hypopituitarism and growth hormone deficiency (GHD) after traumatic brain injury (TBI). Growth Horm IGF Res 2004, 14(Suppl A):S114-S117.
18. Darzy K.H., Shalet S.M. Pathophysiology of radiation-induced growth hormone deficiency: efficacy and safety of GH replacement. Growth Horm IGF Res 2006, 16(Suppl A):S30-S40.
19. Roman J., Villaizan C.J., Garcia-Foncillas J., et al. Growth and growth hormone secretion in children with cancer treated with chemotherapy. J Pediatr 1997, 131:105-112.
20. Blizzard R.M., Bulatovic A. Psychosocial short stature: a syndrome with many variables. Baillieres Clin Endocrinol Metab 1992, 6:687-712.
21. Pandian R., Nakamoto J.M. Rational use of the laboratory for childhood and adult growth hormone deficiency. Clin Lab Med 2004, 24:141-174.
22. Gale E.A., Bennett T., MacDonald I.A., et al. The physiological effects of insulin-induced hypoglycaemia in man: responses at differing levels of blood glucose. Clin Sci 1983, 65:263-271.
23. Shah A., Stanhope R., Matthew D. Hazards of pharmacological tests of growth hormone secretion in childhood. Br Med J 1992, 304:173-174.
24. Frasier S.D. A review of growth hormone stimulation tests in children. Pediatrics 1974, 53:929-937.
25. Bonfig W., Bechtold S., Bachmann S., et al. Reassessment of the optimal growth hormone cut-off level in insulin tolerance testing for growth hormone secretion in patients with childhood-onset growth hormone deficiency during transition to adulthood. J Pediatr Endocrinol Metab 2008, 21:1049-1056.
26. Rosenfeld R.G. Evaluation of growth and maturation in adolescence. Pediatr Rev 1982, 4:175-183.
27. Ranke M.B., Schweizer R., Wollmann H.A., et al. Dosing of growth hormone in growth hormone deficiency. Horm Res 1999, 51(Suppl 3):70-74.
28. Cutfield W., Lindberg A., Albertsson Wikland K., et al. Final height in idiopathic growth hormone deficiency: the KIGS experience. KIGS International Board. Acta Paediatr Suppl 1999, 88:72-75.
29. Wetterau L., Cohen P. Role of insulin-like growth factor monitoring in optimizing growth hormone therapy. J Pediatr Endocrinol Metab 2000, 13(Suppl 6):1371-1376.
30. Zucchini S., Pirazzoli P., Baronio F., et al. Effects on adult height of pubertal growth hormone retesting and withdrawal of therapy in patients with previously diagnosed growth hormone deficiency. J Clin Endocrinol Metab 2006, 91:4271-4276.
31. Giovannuci E., Pollak M. Risk of the cancer after growth hormone treatment. Lancet 2002, 360:258-259.
32. Sklar C.A. Growth hormone treatment: cancer risk. Horm Res 2004, 62:30-34.
33. Seikaly M.G., Salhab N., Gipson D., et al. Stature in children with chronic kidney disease: analysis of NAPRTCS database. Pediatr Nephrol 2006, 21:793-799.
34. Johannsson G., Ahlmen J. End-stage renal disease: endocrine aspects of treatment. Growth Horm IGF Res 2001, 11:59-71.
35. Mehls O., Wuhl E., Tonsshoff B., et al. Growth hormone treatment in short children with chronic kidney disease. Acta Paediatr 2008, 97:1159-1164.
36. Roelfsema V., Clark R.G. The growth hormone and insulin-like growth factor axis: its manipulation for the benefit of growth disorders in renal failure. J Am Soc Nephrol 2001, 12:1297-1306.
37. Tonshoff B., Cronin M.J., Reichert M., et al. Reduced concentration of serum growth hormone (GH)-binding protein in children with chronic renal failure: correlation with GH insensitivity. The European Study Group for Nutritional Treatment of Chronic Renal Failure in Childhood. The German Study Group for Growth Hormone Treatment in Chronic Renal Failure. J Clin Endocrinol Metab 1997, 82:1007-1013.
38. Rabkin R., Sun D.F., Chen Y., et al. Growth hormone resistance in uremia, a role for impaired JAK/STAT signaling. Pediatr Nephrol 2005, 20:313-318.
39. Nissel R., Lindberg A., Mehls O., et al. Pfizer International Growth Database (KIGS) International Board. Factors predicting the near final height in growth hormone-treated children and adolescents with chronic kidney disease. J Clin Endocrinol Metab 2008, 93:1359-1365.
40. Wuhl E., Schaefer F. Effects of growth hormone in patients with chronic renal failure: experience in children and adults. Horm Res 2002, 58(Suppl 3):35-38.
41. Doi T., Striker L.J., Quaife C., et al. Progressive glomerulosclerosis develops in transgenic mice chronically expressing growth hormone and growth hormone-releasing factor but not in those expressing insulin-like growth factor-1. Am J Pathol 1988, 131:398-403.
42. Fine R.N., Sullivan E.K., Kuntze J., et al. The impact of recombinant human growth hormone treatment during chronic renal insufficiency on renal transplant recipients. J Pediatr 2000, 136:376-382.
43. Guest G., Berard E., Crosnier H., et al. Effects of growth hormone in short children after renal transplantation. French Society of Pediatric Nephrology. Pediatr Nephrol 1998, 12:437-446.
44. Cutfield W.S., Wilton P., Bennmarker H., et al. Incidence of diabetes mellitus and impaired glucose tolerance in children and adolescents receiving growth hormone treatment. Lancet 2000, 355:610-613.
45. Ranke M.B., Pfluger H., Rosendahl W., et al. Turner syndrome: spontaneous growth in 150 cases and review of the literature. Eur J Pediatr 1983, 141:81-88.
46. Rao E., Weiss B., Fukami M., et al. Pseudoautosomal deletions encompassing a novel homeobox gene cause growth failure in idiopathic short stature and Turner syndrome. Nat Genet 1997, 16:54-63.
47. Davenport ML, Quigley CA, Bryant CG, et al. Effect of early growth hormone (GH) treatment in very young girls with Turner syndrome (TS). Presented at the Seventh Joint European Society for Paediatric Endocrinology/Lawson Wilkins Pediatric Endocrine Society Meeting. Lyon, France, September 23, 2005.
48. Rosenfeld R.G., Attie K.M., Frane J., et al. Growth hormone therapy of Turner's syndrome: beneficial effect on adult height. J Pediatr 1998, 132:319-324.
49. Bolar K., Hoffman A.R., Maneatis T., et al. Long-term safety of recombinant human growth hormone in turner syndrome. J Clin Endocrinol Metab 2008, 93:344-351.
50. Munce T., Simpson R., Bowling F. Molecular characterization of Prader-Willi syndrome by real-time PCR. Genet Test 2008, 12:319-324.
51. Tauber M. Effects of growth hormone treatment in children presenting with Prader-Willi syndrome: the KIGS experience. Growth hormone therapy in Pediatrics-20 years of KIGS 2007, 377-387. Karger, Basel. M.B. Ranke, D.A. Price, E.O. Reiter (Eds.).
52. Wollmann H.A., Schultz U., Grauer M.L., et al. Reference values for height and weight in Prader-Willi syndrome based on 315 patients. Eur J Pediatr 1998, 157:634-642.
53. Corrias A., Bellone J., Beccaria L., et al. IGF-I axis in Prader-Willi syndrome: evaluation of IGF-I levels and of the somatotroph responsiveness to various provocative stimuli. Genetic Obesity Study Group of Italian Society of Pediatric Endocrinology and Diabetology. J Endocrinol Invest 2000, 23:84-89.
54. Eiholzer U., Bachmann S., I'Allemand D. Is there growth hormone deficiency in Prader-Willi syndrome? Six arguments to support the presence of hypothalamic growth hormone deficiency in Prader-Willi syndrome. Horm Res 2000, 53(Suppl 3):44-52.
55. Carrel A.L., Myers S.E., Whitman B.Y., et al. Benefits of long-term GH therapy in Prader-Willi syndrome: a 4-year study. J Clin Endocrinol Metab 2002, 87:1581-1585.
56. Angulo M.A., Castro-Magana M., Lamerson M., et al. Final adult height in children with Prader-Willi syndrome with and without human growth hormone treatment. Am J Med Genet A 2007, 143:1456-1461.
57. Whitman B.Y., Myers S., Carrel A., et al. The behavioral impact of growth hormone treatment for children and adolescents with Prader-Willi syndrome: a 2-year, controlled study. Pediatrics 2002, 109:E35.
58. Festen D.A.M., Wevers M., Lindgren A.C., et al. Mental and motor development before and during growth hormone treatment in infants and toddlers with Prader-Willi syndrome. Clin Endocrinol (Oxf) 2008, 68:919-925.
59. Festen D.A., Visser T.J., Otten B.J., et al. Thyroid hormone levels in children with Prader-Willi syndrome before and during growth hormone treatment. Clin Endocrinol (Oxf) 2007, 67:449-456.
60. Tauber M., Diene G., Molinas C., et al. A review of 64 cases of death in children with Prader-Willi syndrome (PWS). Am J Med Genet A 2008, 46:881-887.
61. Mogul H.R., Lee P.D., Whitman B.Y., et al. Growth hormone treatment of adults with Prader-Willi syndrome and growth hormone deficiency improves lean body mass, fractional body fat, and serum triiodothyronine without glucose impairment: results from the United States multicenter trial. J Clin Endocrinol Metab 2008, 93:1238-1245.
62. Hokken-Koelega A.C., De Ridder M.A., Lemmen R.J., et al. Children born small for gestational age: do they catch up?. Pediatr Res 1995, 38:267-271.
63. Lee P.A., Chernausek S.D., Hokken-Koelega A.C. International Small for Gestational Age Advisory Board consensus development conference statement: management of short children born small for gestational age, April 24-October 1, 2001. Pediatrics 2003, 111:1253-1261.
64. Sas T., Mulder P., Aanstoot H.J., et al. Carbohydrate metabolism during long-term growth hormone treatment in children with short stature born small for gestational age. Clin Endocrinol (Oxf) 2001, 54:243-251.
65. Ranke M.B. Towards a consensus on the definition of idiopathic short stature. Horm Res 1996, 45(Suppl 2):64-66.
66. Cohen P., Rogol A.D., Deal C.L., 2007 Consensus Workshop participants, et al. Consensus statement on the diagnosis and treatment of children with idiopathic short stature: a summary of the Growth Hormone Research Society, the Lawson Wilkins Pediatric Endocrine Society, and the European Society for Paediatric Endocrinology Workshop. J Clin Endocrinol Metab 2008, 93:4210-4217.
67. Rochiccioli P., Battin J., Bertrand A.M., et al. Final height in Turner syndrome patients treated with growth hormone. Horm Res 1995, 44:172-176.
68. Cohen P., Rogol A.D., Howard C.P., et al. Insulin growth factor-based dosing of growth hormone therapy in children: a randomized, controlled study. J Clin Endocrinol Metab 2007, 92:2480-2486.
69. Belin V., Cusin V., Viot G., et al. SHOX mutations in dyschondrosteosis (Léri-Weill syndrome). Nat Genet 1998, 19:67-69.
70. Huber C., Rosilio M., Munnich A., French SHOX GeNeSIS Module, et al. High incidence of SHOX anomalies in patients with short stature. J Med Genet 2006, 43:735-739.
71. Blum W.F., Crowe B.J., Quigley C.A., et al. Growth hormone is effective in treatment of short stature associated with short stature homeobox-containing gene deficiency: two-year results of a randomized, controlled multicenter trial. J Clin Endocrinol Metab 2007, 92:219-228.
72. Tartaglia M., Mehler E.L., Goldberg R., et al. Mutations in PTPN11, encoding the protein tyrosine phosphatase SHP-2, cause Noonan syndrome. Nat Genet 2001, 29:465-468.
73. Stofega M.R., Herrington J., Billestrup N., et al. Mutation of the SHP-2 binding site in growth hormone (GH) receptor prolongs GH-promoted tyrosyl phosphorylation of GH receptor, JAK2, and STAT5B. Mol Endocrinol 2000, 14:1338-1350.
74. 3 integrin regulates insulin-like growth factor I (IGF-I) receptor phosphorylation by altering the rate of recruitment of the Src-homology 2-containing phosphotyrosine phosphatase-2 to the activated IGF-I receptor. Endocrinology 2002, 143:4259-4264.
75. Ferreira L.V., Souza S.A., Arnhold I.J., et al. PTPN11 mutations and response to growth hormone therapy in children with Noonan syndrome. J Clin Endocrinol Metab 2005, 90:5156-5160.
76. 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.
77. Cotterill A.M., McKenna W.J., Brady A.F., et al. The short-term effects of growth hormone therapy on height velocity and cardiac ventricular wall thickness in children with Noonan's syndrome. J Clin Endocrinol Metab 1996, 81:2291-2297.
78. Molitch M.E., Clemmons D.R., Malozowski S., et al. Endocrine Society's Clinical Guidelines Subcommittee. Evaluation and treatment of adult growth hormone deficiency: an Endocrine Society clinical practice guideline. J Clin Endocrinol Metab 2006, 91:1621-1634.
79. Laursen T., Jørgensen J.O., Christiansen J.S. The management of adult growth hormone deficiency syndrome. Expert Opin Pharmacother 2008, 9:2435-2450.
80. Biller B.M., Samuels M.H., Zagar A., et al. Sensitivity and specificity of six tests for the diagnosis of adult GH deficiency. J Clin Endocrinol Metab 2002, 87:2067-2079.
81. Mulligan K., Schambelan M. Anabolic treatment with GH, IGF-I, or anabolic steroids in patients with HIV-associated wasting. Int J Cardiol 2002, 85:151-159.
82. Morley J.E., Thomas D.R., Wilson M.M. Cachexia: pathophysiology and clinical relevance. Am J Clin Nutr 2006, 83:735-743.
83. Heijligenberg R., Sauerwein H.P., Brabant G., et al. Circadian growth hormone secretion in asymptomatic human immune deficiency virus infection and acquired immunodeficiency syndrome. J Clin Endocrinol Metab 1996, 81:4028-4032.
84. Geffner M.E., Yeh D.Y., Landaw E.M., et al. In vitro insulin-like growth factor-I, growth hormone, and insulin resistance occurs in symptomatic human immunodeficiency virus-1-infected children. Pediatr Res 1993, 34:66-72.
85. Schambelan M., Mulligan K., Grunfeld C., et al. Recombinant human growth hormone in patients with HIV-associated wasting. A randomized, placebo-controlled trial. Serostim Study Group. Ann Intern Med 1996, 125:873-882.
86. Gelato M., McNurlan M., Freedland E. Role of recombinant human growth hormone in HIV-associated wasting and cachexia: pathophysiology and rationale for treatment. Clin Ther 2007, 29:2269-2288.
87. Macallan D.C., Baldwin C., Mandalia S., et al. Treatment of altered body composition in HIV-associated lipodystrophy: comparison of rosiglitazone, pravastatin, and recombinant human growth hormone. HIV Clin Trials 2008, 9:254-268.
88. Lund P.K., Ulshen M.H., Rountree D.B., et al. Molecular biology of gastrointestinal peptides and growth factors: relevance to intestinal adaptation. Digestion 1990, 46(Suppl 2):66-73.
89. Guarino A., De Marco G., Italian National Network for Pediatric Intestinal Failure Natural history of intestinal failure, investigated through a national network-based approach. J Pediatr Gastroenterol Nutr 2003, 37:136-141.
90. Pereira P.M., Bines J.E. New growth factor therapies aimed at improving intestinal adaptation in short bowel syndrome. J Gastroenterol Hepatol 2006, 21:932-940.
91. Messing B., Blethen S., Dibaise J.K., et al. Treatment of adult short bowel syndrome with recombinant human growth hormone: a review of clinical studies. J Clin Gastroenterol 2006, 40(Suppl 2):S75-S84.
92. Weiming Z., Ning L., Jieshou L. Effect of recombinant human growth hormone and enteral nutrition on short bowel syndrome. JPEN J Parenter Enteral Nutr 2004, 28:377-381.