Consensus
Linee guida sulla diagnosi e il trattamento del bambino con bassa statura idiopatica
GUIDELINES ON DIAGNOSIS AND TREATMENT OF CHILDREN WITH IDIOPATHIC SHORT STATURE
Francesco Chiarelli*, Rita Salomone
Clinica Pediatrica, Universitŕ di Chieti
*Presidente della Societŕ Europea di Endocrinologia Pediatrica (ESPE)
Dicembre 2008 - pagg. 626 -633
Abstract
ISS is defined auxologically by a height below -2 SDS without findings of disease after a complete evaluation by a pediatric endocrinologist (including bone age X-ray, thyroid function, karyotype for girls, and measurements of IGF-I and stimulated growth hormone levels). This definition includes children with “constitutional delay of growth and puberty
(CDGP)” and “familial short stature”. According to experts the height criteria to assess therapy
vary between -2 and -3 SDS. Oxandrolone and low dose testosterone therapy promote the short-term acceleration of growth with, generally, no decrease in adult height potential, and low dose testosterone is the appropriate therapy for males with mild short stature (> -2.5 SDS) and CDGP. GnRH analogue monotherapy is not recommended in children
with ISS. Aromatase inhibition increases predicted adult height in males with ISS, but
adult height data are not available. This therapy is currently not recommended for females.
Psychological counselling is worthwhile to consider instead of or as an adjunct to
hormone treatment. The shorter the child, the more consideration should be given to treatment
with GH. Responses are highly variable and depend on dose, length of therapy
and other, but presently unknown, factors. IGF-I levels may be helpful in assessing compliance and GH sensitivity. If height prediction is below -2 SDS at the time of pubertal onset in either sex, the addition of GnRH analogues may be considered. GH therapy for children with ISS has a similar safety profile to other GH indications.
Classificazione MeSH
Bibliografia
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• Visser-van Balen H, Geenen R, Kamp GA, Huisman J, Wit JM, Sinnema G. Long-term psychosocial consequences of hormone treatment for short stature. Acta Paediatr 2007; 96:715-9.
• Wit JM, Clayton PE, Rogol AD, Saenger PH, Savage MO, Cohen P. Idiopathic short stature: definition, epidemiology, and diagnostic evaluation. Growth Horm IGF Res 2008; 18:89-110.
• Wit JM, Reiter E, Ross JL, et al. Idiopathic short stature: management and growth hormone treatment. Growth Horm IGF Res 2008;18: 111-35.
• Wit JM, Rekers-Mombarg LT, Cutler GB, et al. Growth hormone (GH) treatment to final height in children with idiopathic short stature: evidence for a dose effect. J Pediatr 2005; 146:45-53.
• Cohen P, Rogol AD, Howard CP, Bright GM, Kappelgaard AM, Rosenfeld RG. Effects of IGFbased dosing on the efficacy of GH therapy in children: results of a randomized concentrationcontrolled study. J Clin Endocrinol Metab 2007; 92:2480-6.
• Consensus guidelines for the diagnosis and treatment of adults with growth hormone deficiency: summary statement of the Growth Hormone Research Society Workshop on Adult Growth Hormone Deficiency. J Clin Endocrinol Metab 1998;83:379-81.
• Consensus guidelines for the diagnosis and treatment of growth hormone (GH) deficiency in childhood and adolescence: summary statement of the GH Research Society. J Clin Endocrinol Metab 2000;85:3990-3.
• Finkelstein BS, Imperiale TF, Speroff T, Marrero U, Radcliffe DJ, Cuttler L. Effect of growth hormone therapy on height in children with idiopathic short stature: a meta-analysis. Arch Pediatr Adolesc Med 2002;156:230-40.
• Hero M, Wickman S, Dunkel L. Treatment with the aromatase inhibitor letrozole during adolescence increases near-final height in boys with constitutional delay of puberty. Clin Endocrinol 2006;64:510-3.
• Hintz RL, Attie KM, Baptista J, Roche A. Effect of growth hormone treatment on adult height of children with idiopathic short stature. Genentech Collaborative Group. N Engl J Med 1999;340:502-7.
• Leschek EW, Rose SR, Yanovski JA, et al.; National Institute of Child Health and Human Development- Eli Lilly & Co. Growth Hormone Collaborative Group. Effect of growth hormone treatment on adult height in peripubertal children with idiopathic short stature: a randomized, double-blind, placebo-controlled trial. J Clin Endocrinol Metab 2004;89:3140-8.
• Quigley CA, Gill AM, Crowe BJ, et al. Safety of growth hormone treatment in pediatric patients with idiopathic short stature. J Clin Endocrinol Metab 2005;90:5188-96.
• Ranke MB, Lindberg A, Price DA, et al.; 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 2007;68:53-62.
• Rappold G, Blum WF, Shavrikova EP, et al. Genotypes and phenotypes in children with short stature: clinical indicators of SHOX haploinsufficiency. J Med Genet 2007;44:306-13.
• Ross JL, Sandberg DE, Rose SR, et al. Psychological adaptation in children with idiopathic short stature treated with growth hormone or placebo. J Clin Endocrinol Metab 2004; 89:4873-8.
• van Gool SA, Kamp GA, Visser-van Balen H, et al. Final height outcome after three years of growth hormone and gonadotropin-releasing hormone agonist treatment in short adolescents with relatively early puberty. J Clin Endocrinol Metab 2007;92:1402-8.
• Visser-van Balen H, Geenen R, Kamp GA, Huisman J, Wit JM, Sinnema G. Long-term psychosocial consequences of hormone treatment for short stature. Acta Paediatr 2007; 96:715-9.
• Wit JM, Clayton PE, Rogol AD, Saenger PH, Savage MO, Cohen P. Idiopathic short stature: definition, epidemiology, and diagnostic evaluation. Growth Horm IGF Res 2008; 18:89-110.
• Wit JM, Reiter E, Ross JL, et al. Idiopathic short stature: management and growth hormone treatment. Growth Horm IGF Res 2008;18: 111-35.
• Wit JM, Rekers-Mombarg LT, Cutler GB, et al. Growth hormone (GH) treatment to final height in children with idiopathic short stature: evidence for a dose effect. J Pediatr 2005; 146:45-53.
Corrispondenza: chiarell@unich.it