Growth response to rhIGF-I 80 μg/kg twice daily in children with growth hormone insensitivity syndrome: Relationship to severity of clinical phenotype

Clinical Endocrinology

Volumn 51, Issue 6, 1999, Pages 787-792

  Azcona, Cristina ^a   Preece, Michael A ^b   Rose, Stephen J ^d   Fraser, Neil ^d   Rappaport, Raphaël ^c   Ranke, Michael B ^d   Savage, Martin O ^a,b  

^a ST BARTHOLOMEW S HOSPITAL   (United Kingdom)

^b GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^c HÔPITAL NECKER ENFANTS MALADES   (France)

^d NONE

Author keywords

[No Author keywords available]

Indexed keywords

GROWTH HORMONE; SOMATOMEDIN C;

ARTICLE; BODY HEIGHT; BODY MASS; BONE AGE; CHILD; CLINICAL ARTICLE; DISEASE SEVERITY; DRUG EFFICACY; DRUG SAFETY; FEMALE; GROWTH; HEADACHE; HORMONE RESISTANCE; HUMAN; HYPOGLYCEMIA; LIPOHYPERTROPHY; MALE; PAPILLEDEMA; PRIORITY JOURNAL;

BODY HEIGHT; CHILD; CHILD, PRESCHOOL; DRUG ADMINISTRATION SCHEDULE; FEMALE; GROWTH DISORDERS; GROWTH HORMONE; HEADACHE; HUMANS; INJECTIONS, SUBCUTANEOUS; INSULIN-LIKE GROWTH FACTOR BINDING PROTEIN 3; INSULIN-LIKE GROWTH FACTOR I; MALE; PAIN; PAPILLEDEMA; RECOMBINANT PROTEINS;

EID: 0033393584     PISSN: 03000664     EISSN: None     Source Type: Journal    
DOI: 10.1046/j.1365-2265.1999.00887.x     Document Type: Article

References (28)

1. Amselem, S., Sobrier, M.L., Dastot, F., Duquesnoy, P., Duriez, B. & Goossens, M. (1996) Molecular basis of inherited growth hormone resistance in childhood. In: Growth Hormone Resistance (eds. R.J.M. Ross & M.O. Savage), pp. 353-370. Baillière-Tindall, London.
2. Backeljauw, P.F. & Underwood, L.E. (1996) Prolonged treatment with recombinant insulin-like growth factor-I in children with growth hormone insensitivity syndrome - a clinical research center study. GHIS Collaborative Group. Journal of Clinical Endocrinology and Metabolism, 81, 3312-3317.
3. Baumann, G., Shaw, M.A. & Winter, R.J. (1987) Absence of the growth hormone-binding protein in Laron-type dwarfism. Journal of Clinical Endocrinology and Metabolism, 65, 814-816.
4. Blum, W.F., Ranke, M.B., Savage, M.O. & Hall, K. (1992) Insulin like growth factors and their binding proteins in patients with growth hormone receptor deficiency: suggestions for new criteria. Acta Paediatrica, 383 (suppl.), 125-126.
5. Blum, W.F., Cotterill, A.M., Postel-Vinay, M.C., Ranke, M.B., Savage, M.O. & 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 Paediatrica, 399 (suppl.), 117-124.
6. Cameron, N. (1978) The methods of auxological anthropometry. In: Human Growth (eds. F. Falkner & J.M. Tanner), pp. 35-87. Plenum, New York.
7. Gargosky, S.E., Wilson, K.F. & Fielder, P.J. (1993) The composition and distribution of insulin-like growth factors (IGFs) and IGF-binding proteins (IGFBPs) in the serum of growth hormone receptor-deficient patients: effects of IGF-I therapy on IGFBP-3. Journal of Clinical Endocrinology and Metabolism, 77, 1683-1689.
8. Gluckman, P. (1994) Growth hormone deficiency diagnosed and treated in the first two years of life: evidence of the role of growth hormone in human perinatal growth. In: Progress in Growth Hormone Therapy-5 years of KIGS (eds M. Ranke & R. Gunnarson), 88-96. J & J Verlag, Mannheim.
9. Guevara-Aguirre, J., Vasconez, O., Martinez, V., Martinez, A.L., Rosembloom, A.L., Diamond, F.B., Gargosky, S.E., Nonoshita, L. & Rosenfeld, R.G. (1995) A randomized, double blind, placebo-controlled trial on safety and efficacy of recombinant human insulin-like growth factor-I in children with growth hormone receptor deficiency. Journal of Clinical Endocrinology and Metabolism, 80, 1393-1398.
10. Guevara-Aguirre, J., Rosenbloom, A.L., Vasconez, O., Martinez, V., Gargosky, S.E., Allen, L. & Rosenfeld, R.G. (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. Journal of Clinical Endocrinology and Metabolism, 82, 629-633.
11. Heinrichs, C., Vis, H.L., Bergmann, P., Wilton, P. & Bourguignon, J.P. (1993) Effects of 17 months treatment using recombinant insulin-like growth factor-I in two children with growth hormone insensitivity (Laron) syndrome. Clinical Endocrinology (Oxf), 38, 647-651.
12. Klinger, B. & Laron, Z. (1995) Three year IGF-I treatment of children with Laron syndrome. Journal of Pediatric Endocrinology and Metabolism, 8, 149-158.
13. Labarta, J.I., Gargosky, S.E., Simpson, D.M., Lee, P.D.K., Argente, J., Guevara-Aguirre, J. & Rosenfeld, R.G. (1997) Immunoblot studies of the acid-labile subunit (ALS) in biological fluid, normal human serum and in children with GH deficiency and GH receptor deficiciency before and after long-term therapy with GH or IGF-I respectively. Clinical Endocrinology, 47, 657-666.
14. Laron, Z., Pertzelan, A. & Mannheimer, S. (1966) Genetic pituitary dwarfism with high serum concentration of growth hormone. A new inborn error of metabolism? Israeli Journal of Medicine and Science, 2, 152-155.
15. Laron, Z., Anin, S., Klipper-Aurbach, Y. & Klinger, B. (1992) Effects of insulin-like growth factor on linear growth, head circumference, and body fat in patients with Laron-type dwarfism. Lancet, 339, 1258-1261.
16. Laron, Z., Lilos, P. & Klinger, B. (1993) Growth curves for Laron syndrome. Archives of Disease in Childhood, 68, 2768-2770.
17. Lorderau-Richard, I., Roger, M. & Chaussain, J.L. (1994) Transient papilloedema in a 10-year-old boy treated with recombinant insulin-like growth factor I for growth hormone receptor deficiency. Acta Paediatrica, 399 (suppl.), 153.
18. Malozowski, S., Tanner, L., Wysowski, D. & Fleming, G. (1993) Growth hormone, insulin-like growth factor 1, and benign intracranial hypertension. New England Journal of Medicine, 329, 65-66.
19. Ranke, M.B., Savage, M.O., Chatelain, P.G., Preece, M.A., Rosenfeld, R.G., Blum, W.F. & Wilton, P. (1995) Insulin-like growth factor I improves height in growth hormone insensitivity: two years' results. Hormone Research, 44, 253-264.
20. Ranke, M.B. (1996) Treatment of growth hormone insensitivity syndrome (GHIS) with insulin-like growth factor (IGF-I). In: Growth Hormone Resistance (eds R.J.M. Ross & M.O. Savage), pp. 401-410. Baillière-Tindall, London.
21. Rolland-Cachera, M.F., Sempe, M., Guilloud-Bataille, M., Patois, E., Péquignot-Guggenbuhl, M. & Fautrad, V. (1982) Adiposity indices in children. American Journal of Clinical Nutrition, 36, 178-184.
22. Rosenfeld, R.G., Rosenbloom, A.L. & Guevara-Aguirre, J. (1994) Growth hormone (GH) insensitivity due to primary GH receptor deficiency. Endocrine Reviews, 15, 369-390.
23. Savage, M.O., Blum, W.F., Ranke, M.B., Postel-Vinay, M.C., Cotterill, A.M., Hall, K., Chatelain, P.G., Preece, M.A. & Rosenfeld, R.G. (1993) Clinical features and endocrine status in patients with growth hormone insensitivity (Laron syndrome). Journal of Clinical Endocrinology and Metabolism, 77, 1465 -1471.
24. Tanner, J.M. (1962) Growth at Adolescence (ed. J.M. Tanner). Blackwell, Oxford.
25. Tanner, J., Whitehouse, R.H. & Takaishi, M. (1966) Standards from birth to maturity for height, weight, height velocity and weight velocity: British children 1965. Part II. Archives of Disease in Childhood, 41, 454-613.
26. Tanner, J.M., Whitehouse, R.H., Cameron, N., Marshall, W.A., Healy, M.J.R. & Goldstein, H. (1983) Assessment of Skeletal Maturity and Prediction of Adult Height. 2nd edn. Blackwell, Oxford.
27. Woods, K.A., Fraser, N.C., Postel-Vinay, M.C., Savage, M.O. & Clark, A.J. (1996) A homozygous splice site mutation affecting the intracellular domain of the growth hormone (GH) receptor resulting in Laron syndrome with elevated GH-binding protein. Journal of Clinical Endocrinology and Metabolism, 81, 1686-1690.
28. Woods, K.A., Dastot, F., Preece, M.A., Clark, A.J.L., Postel-Vinay, M.C., Chatelain, P.G., Ranke, M.B., Rosenfeld, R.G., Amselem, S. & Savage, M.O. (1997) Phenotype : genotype relationships in growth hormone insensitivity syndrome. Journal of Clinical Endocrinology and Metabolism, 82, 3529-3535.