eMedicine Specialties > Pediatrics: General Medicine > Endocrinology

Short Stature: Treatment & Medication

Author: Robert J Ferry Jr, MD, Chief, Division of Pediatric Endocrinology and Metabolism, Le Bonheur Children's Medical Center, University of Tennessee Health Science Center at Memphis, and St Jude Children's Research Hospital; Field Surgeon (Medical Corps), 162nd Area Support Medical Company, Army National Guard
Contributor Information and Disclosures

Updated: Aug 4, 2009

Treatment

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

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

  • 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.

Medication

Medication administered depends on the etiology of the short stature.

Growth Hormone

These agents improve symptoms associated with growth hormone deficiency (GHD).


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. They did not identify any clinical feature that, prior to start of therapy, could predict whether an individual patient would respond to rhGH and to what degree. 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.

Adult

Pediatric

Regimens vary according to product and the indication
The following doses of recombinant human somatotropin analogs (Humatrope, Nutropin, Nutropin AQ, Saizen) have proven effective
Non-Laron IGFD: 0.05 mg/kg/d SC hs for prepubertal children, 0.1 mg/kg/d SC for pubertal females; 0.2 mg/kg/d SC for pubertal males
TS or chronic renal insufficiency: 0.08 mg/kg/d SC hs
Prader-Willi syndrome: 0.05 mg/kg/d SC

Data limited; several studies suggest that GH may alter clearance of CYP450 substrates

Documented hypersensitivity; active neoplasm; critical illness related to respiratory failure (critical illness due to burns has not been established as contraindication); extreme obesity associated with Prader-Willi syndrome

Pregnancy

C - Safety for use during pregnancy has not been established.

Precautions

Monitor linear growth and serum IGF-I levels at least every 6 months during therapy; antibodies may develop to protein contained in drug; monitor blood glucose levels and thyroid levels

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.


Mecasermin (Increlex)

Recombinant human insulinlike growth factor-1 (rhIGF-1) indicated for long-term treatment of GF in children with severe primary IGFD (primary IGFD defined as basal serum IGF-I level and height SD scores <-3, normal or elevated serum GH level). 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.

Adult

Contraindicated

Pediatric

<2 years: Not established
>2 years: 0.04-0.08 mg/kg SC bid initially with meal or snack; if tolerated after 1 wk, may increase by 0.04 mg/kg/dose, not to exceed 0.12 mg/kg bid
Individualize dose and adjust downward if hypoglycemia occurs

Data limited; caution with coadministration of other drugs that alter blood glucose levels

Documented hypersensitivity; closed epiphyses; active or suspected neoplasia; IV administration

Pregnancy

C - Safety for use during pregnancy has not been established.

Precautions

Common adverse effects include hypoglycemia, lipohypertrophy, and tonsillar hypertrophy; contains benzyl alcohol (associated with neurotoxicity in neonates); must be administered with meal or snack to avoid hypoglycemic effect (preprandial glucose monitoring recommended); similar to GH administration, intracranial hypertension with papilledema may develop and cause visual changes, headache, nausea, or vomiting; rapid growth may cause slipped capital femoral epiphysis and scoliosis progression; protein substance administration may cause local or systemic reaction (eg, flushing, hypotension/hypertension, rash, dyspnea); monitor linear growth and serum IGF-I levels every 6 mo while on therapy to maintain IGF-I levels within reference range for Tanner stage and gender while ensuring adequate therapeutic response

More on Short Stature

Overview: Short Stature
Differential Diagnoses & Workup: Short Stature
Treatment & Medication: Short Stature
Follow-up: Short Stature
Multimedia: Short Stature
References
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References

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Further Reading

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Keywords

short stature, short height, familial short stature, genetic short stature, constitutional delay of growth, constitutional growth delay, growth failure, GF, growth pattern, longitudinal growth assessment, chronic short stature, undernutrition genetic disorders, Bayley-Pinneau table, Tanner-Goldstein-Whitehouse table, premature closure of the epiphysial growth plates, growth hormone deficiency, GHD, Turner syndrome, TS, Ullrich-Turner syndrome, eating disorders, malabsorption, polyuria, polydipsia, upper-to-lower segment ratio, US/LS, Hashimoto thyroiditis, pseudohypoparathyroidism, Albright hereditary osteodystrophy, ulcerative stomatitis, Crohn disease, ulcerative colitis, Down syndrome, trisomy 21, insulinlike growth factor, IGF, IGF binding protein, IGFBP, Lerí-Weill dyschondrosteosis, SHOX, treatment, diagnosis

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Contributor Information and Disclosures

Author

Robert J Ferry Jr, MD, Chief, Division of Pediatric Endocrinology and Metabolism, Le Bonheur Children's Medical Center, University of Tennessee Health Science Center at Memphis, and St Jude Children's Research Hospital; Field Surgeon (Medical Corps), 162nd Area Support Medical Company, Army National Guard
Robert J Ferry Jr, MD is a member of the following medical societies: American Academy of Pediatrics, American Diabetes Association, American Medical Association, Endocrine Society, Lawson-Wilkins Pediatric Endocrine Society, Society for Pediatric Research, and Texas Pediatric Society
Disclosure: Nutropin Speakers Bureau Honoraria Speaking and teaching; Genotropin Speakers Bureau Honoraria Speaking and teaching; Eli Lilly & Co. Grant/research funds Independent contractor; MacroGenics, Inc. Grant/research funds Independent contractor; Ipsen, S.A. (formerly Tercica, Inc.) Grant/research funds Independent contractor

Medical Editor

Angelo P Giardino, MD, PhD, Clinical Associate Professor, Department of Pediatrics, Baylor College of Medicine; Medical Director, Texas Children's Health Plan, Inc
Angelo P Giardino, MD, PhD is a member of the following medical societies: Academic Pediatric Association, American Academy of Pediatrics, American Professional Society on the Abuse of Children, Harris County Medical Society, Helfer Society, and International Society for Prevention of Child Abuse and Neglect
Disclosure: Nothing to disclose.

Pharmacy Editor

Mary L Windle, PharmD, Adjunct Assistant Professor, University of Nebraska Medical Center College of Pharmacy, Pharmacy Editor, eMedicine
Disclosure: Pfizer Inc Stock Investment from financial planner; Avanir Pharma Stock Investment from financial planner ; WebMD Salary and stock Employment and investment from financial planner

Managing Editor

Lynne Lipton Levitsky, MD, Chief, Pediatric Endocrine Unit, Massachusetts General Hospital; Associate Professor, Department of Pediatrics, Harvard University Medical School
Lynne Lipton Levitsky, MD is a member of the following medical societies: Alpha Omega Alpha, American Academy of Pediatrics, American Diabetes Association, American Pediatric Society, Endocrine Society, Lawson-Wilkins Pediatric Endocrine Society, and Society for Pediatric Research
Disclosure: Pfizer Grant/research funds P.I.; Tercica Grant/research funds PI, also occasional consultant

CME Editor

Merrily P M Poth, MD, Professor, Department of Pediatrics and Neuroscience, Uniformed Services University of the Health Sciences
Merrily P M Poth, MD is a member of the following medical societies: American Academy of Pediatrics, Endocrine Society, and Lawson-Wilkins Pediatric Endocrine Society
Disclosure: Nothing to disclose.

Chief Editor

Stephen Kemp, MD, PhD, Professor, Department of Pediatrics, Section of Pediatric Endocrinology, University of Arkansas and Arkansas Children's Hospital
Stephen Kemp, MD, PhD is a member of the following medical societies: American Academy of Pediatrics, American Association of Clinical Endocrinologists, American Pediatric Society, Endocrine Society, Phi Beta Kappa, Southern Medical Association, and Southern Society for Pediatric Research
Disclosure: Genentech, Inc. Honoraria Speaking and teaching; Pfizer, Inc. Honoraria Consulting

 
 
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