Short Stature Treatment & Management
- Author: Robert J Ferry Jr, MD; Chief Editor: Stephen Kemp, MD, PhD more...
Medical Care
Medical care depends on the etiology of the short stature.
Recombinant human growth hormone (rhGH) administration has not been proven to remarkably improve final adult height in children with normal variant short stature.[3, 4] Nine inconclusive clinical studies that focused on this particular issue have been published to date. Published studies were flawed because of the following:
- Selection bias due to high drop-out rates from treatment regimens (presumably due, in part, to the parents' or health care provider's dissatisfaction with results of therapy in these individuals)
- Lack of key design elements for a proper clinical trial (eg, placebo controls, double blinding, randomization)
- Inadequate follow-up study to final adult height
A study from the National Institutes of Health was double blinded randomly and suggests GH has a small effect on adult height in children with normal short stature if they are treated with GH injections for many years. In the absence of better clinical outcomes, do not use rhGH therapy to treat children with normal variant short stature. A 2-year, open-label, randomized trial measured the response to somatotropin (rDNA orign) therapy based on serum insulinlike growth factor (IGF)-I levels in children with growth hormone deficiency (GHD) and in children with idiopathic short stature.[5] These data suggest that IGF-based dosing of GH may safely provide superior growth outcome in both groups.
Surgical Care
Surgical care depends on the underlying cause of short stature. Brain tumors that cause hyposomatotropism may require neurosurgical intervention, depending on the tumor type and location (see Hyposomatotropism). Limb-lengthening procedures have been performed but carry enormous morbidity and mortality risks and are not recommended.
Consultations
The following consultations are indicated:
- Consult a pediatric cardiologist, radiologist, and audiologist for patients with Ullrich-Turner syndrome.
- Consult a psychologist for patients with eating disorders.
- Perform all GH provocative testing under the supervision of a pediatric endocrinologist.
Diet
Optimize nutrition in patients with GI disease. Obtain psychologic or psychiatric consultation for patients with eating disorders. Forced energy intake in children with normal variant short stature has not been demonstrated to improve short-term growth or final adult height.
Activity
Do not restrict activity in children with normal variant short stature.
[Best Evidence] [Guideline] Cohen P, Rogol AD, Deal CL, 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. Nov 2008;93(11):4210-7. [Medline].
Lindsay R, Feldkamp M, Harris D, Robertson J, Rallison M. Utah Growth Study: growth standards and the prevalence of growth hormone deficiency. J Pediatr. Jul 1994;125(1):29-35. [Medline].
Albertsson-Wikland K, Aronson AS, Gustafsson J, et al. Dose-dependent effect of growth hormone on final height in children with short stature without growth hormone deficiency. J Clin Endocrinol Metab. Nov 2008;93(11):4342-50. [Medline].
Collett-Solberg PF, Misra M,. The role of recombinant human insulin-like growth factor-I in treating children with short stature. J Clin Endocrinol Metab. Jan 2008;93(1):10-8. [Medline].
Cohen P, Germak J, Rogol AD, et al. Variable Degree of Growth Hormone (GH) and Insulin-Like Growth Factor (IGF) Sensitivity in Children with Idiopathic Short Stature Compared with GH-Deficient Patients: Evidence from an IGF-Based Dosing Study of Short Children. J Clin Endocrinol Metab. Mar 5 2010;[Medline].
Hagman A, Wennerholm UB, Kallen K, et al. Women who gave birth to girls with Turner syndrome: maternal and neonatal characteristics. Hum Reprod. Apr 10 2010;[Medline].
Attie KM, Julius JR, Stoppani C, Rundle AC. National Cooperative Growth Study substudy VI: the clinical utility of growth-hormone-binding protein, insulin-like growth factor I, and insulin-like growth factor-binding protein 3 measurements. J Pediatr. Jul 1997;131(1 Pt 2):S56-60. [Medline].
[Best Evidence] Badaru A, Wilson DM. Alternatives to growth hormone stimulation testing in children. Trends Endocrinol Metab. Aug 2004;15(6):252-8. [Medline].
Bayley N, Pinneau SR. Tables for predicting adult height from skeletal age: revised for use with the Greulich-Pyle hand standards. J Pediatr. Apr 1952;40(4):423-41. [Medline].
Belin V, Cusin V, Viot G, et al. SHOX mutations in dyschondrosteosis (Leri-Weill syndrome). Nat Genet. May 1998;19(1):67-9. [Medline].
Boguszewski CL, Carlsson B, Carlsson LM. Mechanisms of growth failure in non-growth-hormone deficient children of short stature. Horm Res. 1997;48 Suppl 4:19-22. [Medline].
Cohen P, Bright GM, Rogol AD, et al. 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. Jan 2002;87(1):90-8. [Medline]. [Full Text].
de Mel T, Warnasooriya N, Fonseka C. Growth hormone deficiency in Sri Lanka: a preliminary study. Ceylon Med J. Sep 1991;36(3):95-7. [Medline].
Elsas LJ, Endo F, Strumlauf E, Elders J, Priest JH. Leprechaunism: an inherited defect in a high-affinity insulin receptor. Am J Hum Genet. Jan 1985;37(1):73-88. [Medline].
Ferry RJ Jr, Cohen P. The insulin-like growth factor axis in pediatrics. Clin Pediatr Endocrinol. 1999;8(1):1-10.
Gandrud LM, Wilson DM. Is growth hormone stimulation testing in children still appropriate?. Growth Horm IGF Res. Jun 2004;14(3):185-94. [Medline].
Gatta V, Antonucci I, Morizio E, et al. Identification and characterization of different SHOX gene deletions in patients with Leri-Weill dyschondrosteosys by MLPA assay. J Hum Genet. 2007;52(1):21-7. [Medline].
GH Research Society. 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. Nov 2000;85(11):3990-3. [Medline]. [Full Text].
Guyda HJ. Four decades of growth hormone therapy for short children: what have we achieved?. J Clin Endocrinol Metab. Dec 1999;84(12):4307-16. [Medline]. [Full Text].
Guyda HJ. Growth hormone testing and the short child. Pediatr Res. Nov 2000;48(5):579-80. [Medline].
Hardin DS, Woo J, Butsch R, Huett B. Current prescribing practices and opinions about growth hormone therapy: results of a nationwide survey of paediatric endocrinologists. Clin Endocrinol (Oxf). Jan 2007;66(1):85-94. [Medline].
Harris NS, Crawford PB, Yangzom Y, et al. Nutritional and health status of Tibetan children living at high altitudes. N Engl J Med. Feb 1 2001;344(5):341-7. [Medline]. [Full Text].
Horton WA, Hall JG, Scott CI, Pyeritz RE, Rimoin DL. Growth curves for height for diastrophic dysplasia, spondyloepiphyseal dysplasia congenita, and pseudoachondroplasia. Am J Dis Child. Apr 1982;136(4):316-9. [Medline].
Horton WA, Rotter JI, Rimoin DL, Scott CI, Hall JG. Standard growth curves for achondroplasia. J Pediatr. Sep 1978;93(3):435-8. [Medline].
Hunter AG, Hecht JT, Scott CI Jr. Standard weight for height curves in achondroplasia. Am J Med Genet. Mar 29 1996;62(3):255-61. [Medline].
[Best Evidence] Lee MM. Clinical practice. Idiopathic short stature. N Engl J Med. Jun 15 2006;354(24):2576-82. [Medline].
Li H, Leung SS, Lam PK, et al. Height and weight percentile curves of Beijing children and adolescents 0-18 years, 1995. Ann Hum Biol. Sep-Oct 1999;26(5):457-71. [Medline].
Lyon AJ, Preece MA, Grant DB. Growth curve for girls with Turner syndrome. Arch Dis Child. Oct 1985;60(10):932-5. [Medline].
March of Dimes. Fact sheets for achondroplasia. Available at: http://www.modimes.org. [Full Text].
National Institute of Diabetes & Digestive Kidney Diseases. Fact sheets for patients on growth failure in chronic renal insufficiency. Available at: http://www.niddk.nih.gov. [Full Text].
Palka G, Stuppia L, Guanciali Franchi P, et al. Short arm rearrangements of sex chromosomes with haploinsufficiency of the SHOX gene are associated with Leri-Weill dyschondrosteosis. Clin Genet. Jun 2000;57(6):449-53. [Medline].
Palmer CG, Cronk C, Pueschel SM, et al. Head circumference of children with Down syndrome (0-36 months). Am J Med Genet. Jan 1 1992;42(1):61-7. [Medline].
Parkin JM. Incidence of growth hormone deficiency. Arch Dis Child. Nov 1974;49(11):904-5. [Medline].
Parks JS, Brown MR, Hurley DL, Phelps CJ, Wajnrajch MP. Heritable disorders of pituitary development. J Clin Endocrinol Metab. Dec 1999;84(12):4362-70. [Medline]. [Full Text].
Prader A, Largo RH, Molinari L, Issler C. Physical growth of Swiss children from birth to 20 years of age. First Zurich longitudinal study of growth and development. Helv Paediatr Acta Suppl. Jun 1989;52:1-125. [Medline].
Quigley CA, Gill AM, Crowe BJ, et al. Safety of growth hormone treatment in pediatric patients with idiopathic short stature. J Clin Endocrinol Metab. Sep 2005;90(9):5188-96. [Medline].
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. May 1997;16(1):54-63. [Medline].
Roche AF, Wainer H, Thissen D. The RWT method for the prediction of adult stature. Pediatrics. Dec 1975;56(6):1027-33. [Medline].
Rosenbloom AL, Almonte AS, Brown MR, et al. Clinical and biochemical phenotype of familial anterior hypopituitarism from mutation of the PROP1 gene. J Clin Endocrinol Metab. Jan 1999;84(1):50-7. [Medline]. [Full Text].
Rosenfeld RG. Transition from pediatric to adult care for growth hormone deficiency. J Pediatr Endocrinol Metab. May 2003;16 Suppl 3:645-9. [Medline].
Saenger P. Partial growth hormone insensitivity--idiopathic short stature is not always idiopathic. Acta Paediatr Suppl. Feb 1999;88(428):194-8. [Medline].
Satin-Smith MS, Katz LL, Thornton P, Gruccio D, Moshang T Jr. Arm span as measurement of response to growth hormone (GH) treatment in a group of children with meningomyelocele and GH deficiency. J Clin Endocrinol Metab. Apr 1996;81(4):1654-6. [Medline].
Savendahl L, Davenport ML. Delayed diagnoses of Turner's syndrome: proposed guidelines for change. J Pediatr. Oct 2000;137(4):455-9. [Medline].
Shanske AL, Puri M, Marshall B, Saenger P. Unique deletion in exon 5 of SHOX gene in a patient with idiopathic short stature. Horm Res. 2007;67(2):61-6. [Medline].
Shears DJ, Vassal HJ, Goodman FR, et al. Mutation and deletion of the pseudoautosomal gene SHOX cause Leri-Weill dyschondrosteosis. Nat Genet. May 1998;19(1):70-3. [Medline].
Tanner JM, Goldstein H, Whitehouse RH. Standards for children's height at ages 2-9 years allowing for heights of parents. Arch Dis Child. Dec 1970;45(244):755-62. [Medline].
Toledo C, Alembik Y, Aguirre Jaime A, Stoll C. Growth curves of children with Down syndrome. Ann Genet. 1999;42(2):81-90. [Medline].
Turner's Syndrome Society. Ulrich-Turner syndrome. Available at: http://www.turner-syndrome-us.org/. [Full Text].
Weinzimer SA, Homan SA, Ferry RJ, Moshang T. Serum IGF-I and IGFBP-3 concentrations do not accurately predict growth hormone deficiency in children with brain tumours. Clin Endocrinol (Oxf). Sep 1999;51(3):339-45. [Medline].
Westphal O, Lindberg A. Final height in Swedish children with idiopathic growth hormone deficiency enrolled in KIGS treated optimally with growth hormone. Acta Paediatr. Dec 2008;97(12):1698-706. [Medline].
[Best Evidence] Wilson DM, Frane J. A brief review of the use and utility of growth hormone stimulation testing in the NCGS: do we need to do provocative GH testing?. Growth Horm IGF Res. Jul 2005;15 Suppl A:S21-5. [Medline].
[Best Evidence] [Guideline] Wilson TA, Rose SR, Cohen P, Rogol AD, Backeljauw P, Brown R. Update of guidelines for the use of growth hormone in children: the Lawson Wilkins Pediatric Endocrinology Society Drug and Therapeutics Committee. J Pediatr. Oct 2003;143(4):415-21. [Medline].

