Idiopathic short stature
|Idiopathic short stature|
|Classification and external resources|
Idiopathic short stature (ISS) refers to extreme short stature that does not have a diagnostic explanation (idiopathic designates a condition that is unexplained or not understood) after an ordinary growth evaluation. The term has been in use since at least 1975 without a precise percentile or statistical definition of "extreme".
In 2003 Eli Lilly and Company offered a more precise definition of ISS when the pharmaceutical company submitted clinical trial data to the U.S. Food and Drug Administration (FDA) requesting approval to advertise their brand of growth hormone for the treatment of ISS. They proposed a definition of a height more than 2.25 standard deviations below mean, roughly equal to the shortest 1.2% of the population.
Other researchers have described a cutoff of 2.0 standard deviations.
There is some evidence that hormone treatment may not result in a significant improvement in psychosocial functioning.
It is estimated that it would cost US$100,000 or more  to treat someone, but might only move them from the first percentile to perhaps the 10th. An ethical and economic question is would that just transfer the disadvantages to those in what was previously the "2nd" percentile?
- Sizonenko PC, Rabinovitch A, Schneider P, Paunier L, Wollheim CB, Zahnd G (1975). "Plasma growth hormone, insulin, and glucagon responses to arginine infusion in children and adolescents with idiopathic short stature, isolated growth hormone deficiency, panhypopituitarism, and anorexia nervosa". Pediatr. Res. 9 (9): 733–8. doi:10.1203/00006450-197509000-00010. PMID 1105371.
- Leschek EW, Rose SR, Yanovski JA et al. (2004). "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. 89 (7): 3140–8. doi:10.1210/jc.2003-031457. PMID 15240584.
- Bonioli E, Tarò M, Rosa CL et al. (2005). "Heterozygous mutations of growth hormone receptor gene in children with idiopathic short stature". Growth Horm. IGF Res. 15 (6): 405–10. doi:10.1016/j.ghir.2005.08.004. PMID 16213173.
- Visser-van Balen H, Sinnema G, Geenen R (2006). "Growing up with idiopathic short stature: psychosocial development and hormone treatment; a critical review". Arch. Dis. Child. 91 (5): 433–9. doi:10.1136/adc.2005.086942. PMC 2082749. PMID 16632673.
- Lee JM, Davis MM, Clark SJ, Hofer TP, Kemper AR (2006). "Estimated cost-effectiveness of growth hormone therapy for idiopathic short stature". Arch Pediatr Adolesc Med 160 (3): 263–9. doi:10.1001/archpedi.160.3.263. PMID 16520445.
- Savage MO, Camacho-Hübner C, David A et al. (2007). "Idiopathic short stature: will genetics influence the choice between GH and IGF-I therapy?". Eur. J. Endocrinol. 157 Suppl 1: S33–7. doi:10.1530/EJE-07-0292. PMID 17785695.
- Damiani D, Damiani D (2007). "Pharmacological management of children with short stature: the role of aromatase inhibitors". J Pediatr (Rio J) 83 (5 Suppl): S172–7. doi:10.2223/JPED.1699. PMID 17901908.
- Account of the FDA approval of Humatrope for ISS
- Excellent synopsis of GH use for ISS from the Minnesota Dept of Health
- National Organization of Short Statured Adults