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Fall 2005

Strides Made in Treating Children With Short Stature

Research findings are helping improve the treatment of short stature in children, according to Pinchas Cohen, M.D., director of pediatric endocrinology at Mattel Children’s Hospital at UCLA. “We’re making exciting progress in helping these children,” he notes. Short stature affects 3 percent of the population, and refers to children who measure below the third percentile in height on standard growth charts. Often caused by a lack of normal amounts of growth hormone in the bloodstream, short stature can also have its roots in other medical conditions, such as kidney disease or intestinal malabsorption, whose treatment will generally normalize growth. In some cases, however, short stature has no apparent cause.

Children who lack growth hormone have been treated effectively with growth hormone injections for decades. More recently, it has been found that some short-stature children who are not growth-hormone deficient also respond to growth hormone injections. Those who do not respond to this therapy may benefit from new, alternative hormone therapies, recently approved by the Food and Drug Administration (FDA). Using a synthetic form of the IGF-1 hormone (normally produced by the liver), which mediates growth, these alternative therapies can help children for whom treatment was previously unavailable. Clinical trials are in progress, and Dr. Cohen hopes they will demonstrate advantages over existing treatments.

Notably, a groundbreaking study conducted by Dr. Cohen revealed that changing the dosage of growth hormone from one based on a child’s weight to one targeted to achieve a normal level of the IGF-1 growth factor in the bloodstream has yielded a 50 percent improved growth rate among children. Doctors determine the ideal dosage of growth hormone based on the amount that achieves normal levels of IGF-1 in the blood. Over the course of two years, the investigators saw children grow an additional two inches, over and above the average height increase of two inches that would have resulted from the older dosing methods. “This can make a dramatic difference in the lives of these children,” Dr. Cohen observes.

While some children still thrive socially despite short stature, others have more difficulty coping, Dr. Cohen notes. In these cases, psychological support may also be recommended.

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