Pediatric Indications

Growth hormone deficiency (GHD)

Humatrope is indicated for the long-term treatment of pediatric patients who have growth failure due to an inadequate secretion of normal endogenous growth hormone. Humatrope therapy should not be used for growth promotion in pediatric patients with closed epiphyses.

Lilly's Humatrope was approved by the FDA in 1987 for the treatment of growth failure in children with GHD.

For children diagnosed with GHD, growth hormone may be given. For potential growth benefit, this therapy must begin before the growth plates (epiphyses) at the ends of the long bones have closed, or fused (reached maturity).

Turner syndrome

Humatrope is indicated for the treatment of short stature associated with Turner syndrome in patients whose epiphyses are not closed.

Important Information for Patients with Turner Syndrome – In a randomized, concurrent controlled trial, there was a statistically significant increase in the occurrence of otitis media (43% vs. 26%), ear disorders (18% vs. 5%) and surgical procedures (45% vs. 27%) in patients receiving Humatrope compared with untreated control patients. Other adverse events of special interest to Turner syndrome patients were not significantly different between treatment groups. A similar increase in otitis media was observed in an 18-month placebo-controlled trial. Humatrope is used for the treatment of short stature associated with Turner syndrome as long as the patient has open epiphyses (the growth plates at the ends of the long bones).

Turner syndrome is a genetic condition due to partial or complete absence of one of the two X chromosomes usually present in females. It affects one in every 2,000 to 2,500 females and does not affect males.

Short stature is the most common feature affecting about 95 percent of individuals with Turner syndrome. The average height of affected adult woman without treatment is about 4 feet, 8 inches The other main characteristic is poor development of the ovaries resulting in failure to experience puberty in many girls, and causing infertility in most.

Other physical features present in some individuals include:

• Broad chest with widely spaced nipples that may be inverted
• Stocky appearance
• Narrow, high-arched palate (roof of mouth) and crowded teeth
• Receding jaw
• Thick, shorter neck (in some cases, may have a "webbed" appearance)
• Low hairline at the back of the neck
• Prominent ears
• Arms that turn out at the elbow
• Soft, narrow fingernails and toenails that point upward

Individuals with Turner syndrome may also have increased problems with nonverbal learning. This may result in a learning style that makes verbal learning come more easily, and math or spatial problems more difficult.

Characteristics of this type of learning problem include:

• Difficulty imagining objects in relation to each other (visual-spatial processing), difficulty driving, poor sense of direction, difficulty reading a map
• Trouble appreciating subtle social cues, such as facial expressions (social cognition)
• Problems with nonverbal problem-solving, such as math problems
• Clumsiness (psychomotor problems and poor manual dexterity)

Idiopathic short stature (ISS)

Humatrope is indicated for the long-term treatment of idiopathic short stature, also called non-growth-hormone-deficient short stature, defined by height SDS less than or equal to -2.25, and associated with growth rates unlikely to permit attainment of adult height in the normal range, in pediatric patients whose epiphyses are not closed and for whom diagnostic evaluation excludes other causes associated with short stature that should be observed or treated by other means.

Between 1988 and 2001, Lilly conducted two studies of Humatrope treatment in approximately 300 children with ISS. One study showed that children who received Humatrope injections were on average 1.5 inches taller at final adult height than children who did not receive therapy. The other study showed that children grew significantly faster while receiving Humatrope than before they started taking the medication. For the patients in this study whose final adult height could be measured, Humatrope patients were on average 2 to 3 inches taller than they were predicted to be without Humatrope.

Important Information for Patients with Idiopathic Short Stature – In the placebo-controlled study, the adverse events associated with Humatrope therapy were similar to those observed in other pediatric populations treated with Humatrope. Mean serum glucose level did not change during Humatrope treatment. Mean fasting serum insulin levels increased 10% in the Humatrope treatment group at the end of treatment relative to baseline values but remained within the normal reference range. For the same duration of treatment the mean fasting serum insulin levels decreased by 2% in the placebo group. The incidence of above-range values for glucose, insulin, and HbA1c were similar in the growth hormone and placebo-treated groups. No patient developed diabetes mellitus. Consistent with the known mechanism of growth hormone action, Humatrope-treated patients had greater mean increases, relative to baseline, in serum insulin-like growth factor-I (IGF-I) than placebo-treated patients at each study observation. However, there was no significant difference between the Humatrope and placebo treatment groups in the proportion of patients who had at least one serum IGF-I concentration more than 2.0 SD above the age- and gender-appropriate mean (Humatrope: 9 of 35 patients [26%]; placebo: 7 of 28 patients [25%]).

The adverse events observed in the dose-response study (239 patients treated for 2 years) did not indicate a pattern suggestive of a growth hormone dose effect. Among Humatrope dose groups, mean fasting blood glucose, mean glycosylated hemoglobin, and the incidence of elevated fasting blood glucose concentrations were similar. One patient developed abnormalities of carbohydrate metabolism (glucose intolerance and high serum HbA1c) on treatment.

Click on this ISS patient education handbook link to view an Adobe Acrobat pdf file.

Humatrope can help you successfully treat your patients with GHD, Turner syndrome and ISS.

Where you and your patients may find more information about GHD, Turner syndrome and ISS:

• The Human Growth Foundation – The Human Growth Foundation helps children and adults with disorders related to growth or growth hormone through education, research, support and advocacy. This non-profit organization functions as a resource for individuals with questions about their child's development, or adults with inquiries regarding adult growth hormone deficiency. The Human Growth Foundation has more than 20 educational publications. The foundation's website also provides a list of support groups for parents, teens and adults.
  Toll free number: 800-451-6434
  Internet: www.hgfound.org
  E-mail: hgf1@hgfound.org
• The MAGIC Foundation – The MAGIC Foundation for Children's Growth offers services to families of children with growth problems. These services include parent-to-parent networking opportunities, kids' clubs, medical literature in easy-to-understand terms, and more.
  Toll free number: 800-362-4423
  Internet: www.magicfoundation.org
  E-mail: mary@magicfoundation.org
• The Turner Syndrome Society of the United States – The Turner Syndrome Society of the United States assists girls and women with TS and their families.
  Toll free number: 800-365-9944
  Internet: www.turnersyndrome.org
  E-mail: tssus@turnersyndrome.org

SHOX (short stature homeobox-containing gene) deficiency

Please click to view information about SHOX.