Management and Prognosis

Most cases of short stature due to genetic short stature and constitutional delay.
Generally do not require any treatment unless psychological well-being is affected.
Treatment is directed at the underlying cause of short stature.

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.
o A recent 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.
o In the absence of better clinical outcomes, do not use rhGH therapy to treat children with normal variant short stature.

Medication administered depends on the etiology of the short stature.

Drug Category: Growth Hormone
These agents improve symptoms associated with GHD. Also used in Turner syndrome, Prader Willi syndrome, chronic renal failure, IUGR.

Daily subcutaneous injections. 3 monthly follow up to monitor height/ weight and side effects.

Drug Name: Somatropin (Humatrope, Nutropin, Genotropin, Saizen)
hGH produced via recombinant DNA technology in Escherichia coli; widely available since 1985. Currently, only 1 of the 10 largest reported clinical studies has demonstrated that therapy can increase final adult height in patients with normal variant short stature. This most recent NIH-funded study was randomized, placebo controlled, and took place over 14 y. Investigators demonstrated average gain in height did not exceed 4 cm when rhGH treatment of normal variant short stature began prior to puberty and continued through completion of puberty. Whether several years of daily injections are worth the potential, but not promised, relatively small increase in final adult height remains a personal and individual decision involving the patient, patient's family, and physician.

Drug Category: Insulin-like growth factor-I

Indicated for long-term treatment of severe, primary insulin-like growth factor-I (IGF-I) due to mutations of the growth hormone receptor (GH-R) or GH-R downstream signaling pathways.

Drug Name: Mecasermin (Increlex)
Recombinant human insulinlike growth factor-1 (rhIGF-1) indicated for long-term treatment of GF in children with severe primary IGFD. IGF-I is essential for normal growth of children's bones, cartilage, and organs by stimulating uptake of glucose, fatty acids, and amino acids into tissues. IGF-I is the principal hormone for linear growth and directly mediates GH actions. Primary IGFD is characterized by absent IGF-I production despite normal or elevated GH release.
For severe constitutional delay, a short course of low dose sex hormones can be used to promote pubertal growth.

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.
• Limb-lengthening procedures have been performed but carry enormous morbidity and mortality risks and are not recommended.

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. .

Prognosis
• Individuals with normal variant short stature have an excellent prognosis.
• Treatment of patients with classic GHD with rhGH can be expected to yield a height consistent with genetic potential, provided that therapy is initiated at least 5 years prior to the onset of puberty.
• Treatment of hypothyroidism at least 5 years before the onset of puberty is essential to attain a height consistent with the genetic potential.
• Any chronic illness can reduce the adult height achieved if treatment of the condition is initiated late.

Sources
http://www.emedicine.com/ped/topic2087.htm#section~Treatment
http://www.fmshk.org/database/articles/mb05drcheng.pdf
Lecture Notes: Short Stature: A practical approach and Treatment by Dr YS Choy

Contributed by John Lee