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      Treatment of Growth Hormone Deficiency in Very Young Children

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          Growth hormone (GH) deficiency is a rare disease in very young children and a challenge to the physician in terms of clinical recognition, diagnosis and treatment. Here, we review the available information regarding substitution of GH and other pituitary hormones in this patient group. Our results confirm the severity of the clinical presentation and the rapid loss of height (measured in standard deviation scores) in hypopituitary patients that occurs early in life. GH therapy induces a rapid catch-up of growth and the frequency of injections appears to be a more important determinant than the size of the dose itself. Long-term results are available in only a small percentage of patients, yet they show a favourable auxological outcome, compared with target height or historical height data from patients with severe hypopituitarism. In conclusion, although tremendous progress has been made in providing for those children with early onset and severe hypopituitarism, there is still a long way to go before we can claim that we have completely normalized their condition. In particular, careful assessments of the psychological and neurodevelopmental outcomes will become necessary in order to evaluate the ‘final results’ of endocrine replacement therapies.

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          Most cited references 8

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          Serum levels of insulin-like growth factor (IGF)-binding protein-3 (IGFBP-3) in healthy infants, children, and adolescents: the relation to IGF-I, IGF-II, IGFBP-1, IGFBP-2, age, sex, body mass index, and pubertal maturation

           A Juul (1995)
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            Reference Values for IGF-I throughout Childhood and Adolescence: A Model that Accounts Simultaneously for the Effect of Gender, Age, and Puberty

             C Löfqvist (2001)
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              Endocrinopathies associated with midline cerebral and cranial malformations.

              We systematically reviewed a series of patients (n = 85) with midline cerebral and cranial malformations to correlate the endocrinopathy with the neuroanatomic defect. Midline cleft lip and palate was associated not only with growth hormone deficiency (GHD) but also with diabetes insipidus (DI); holoprosencephaly and optic nerve hypoplasia with absence of the septum pellucidum had a similar incidence of GHD and DI. Optic nerve hypoplasia with absence of the septum pellucidum had the highest incidence of multiple pituitary endocrinopathies and of neonatal hypoglycaemia. Unilateral, although more commonly bilateral, optic nerve hypoplasia was associated with GHD.

                Author and article information

                Horm Res Paediatr
                Hormone Research in Paediatrics
                S. Karger AG
                July 2003
                17 November 2004
                : 60
                : Suppl 1
                : 10-17
                aDepartment of Paediatric Endocrinology and INSERM U561, Groupe hospitalier Cochin-Saint Vincent de Paul and Faculté Cochin, Université Paris V, Paris; bDepartment of Pediatrics, Dijon, France
                71220 Horm Res 2003;60(suppl 1):10–17
                © 2003 S. Karger AG, Basel

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                Page count
                Figures: 4, Tables: 1, References: 35, Pages: 8
                GHD from Birth to 2 Years of Age


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