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. Last Updated: 07/27/2016

Study Questions Use of Growth Drug

WASHINGTON -- Kids don't like being short. They hate those playground nicknames -- "shorty," "pint-sized," "short-stuff." And many say they carry psychological scars into adulthood from being short as a child.

Until 11 years ago, there was little medicine could do to help short children who were otherwise normal. The most common cause of short stature is genetics -- short children are the offspring of short parents.

For a small number of children, reduced stature is a result of the failure of their bodies to produce naturally enough human growth hormone to allow normal growth. In 1958, doctors found a way to treat them with small amounts of the hormone, which was derived from the pituitary glands of cadavers.

But economic laws of supply and demand limited the hormone to those whose stature resulted from hormonal deficiency. The hormone itself was simply too rare and expensive to be given to children who just wanted to grow taller faster.

That changed through genetic engineering, which by 1985 allowed scientists to produce an unlimited supply of human growth hormone in the laboratory much more cheaply.

With plenty of hormone available, doctors increasingly are giving the drug to short but otherwise normal children, a team of medical researchers from Case Western Reserve University School of Medicine in Cleveland reported in the current issue of the Journal of the American Medical Association.

The researchers surveyed pediatric endocrinologists -- the medical subspecialty most likely to treat short children -- and found that 40 percent of the patients receiving human growth hormone did not have a hormone deficiency. Some of those children, however, received human growth hormone for other diseases, such as Turner syndrome (underdeveloped gonads) or kidney insufficiency, in which there are known benefits.

For the rest, however, growth hormone was prescribed largely for psycho-social, not medical, reasons -- often because of pressure from parents who wanted taller children.

The researchers, headed by pediatrician Leona Cutler, questioned whether the use of growth hormone for those children is warranted since there is no scientific proof that hormone-induced growth continues into adulthood. Scientific studies, the report said, "have yielded promising but at times controversial results."

While limited treatment with the hormone increased the rate of growth in short but otherwise normal children by between 40 percent and 90 percent, the researchers said in many cases the growth stopped when the children became adults and the treatment was stopped. In effect, while doses of growth hormone allowed them to be normal-sized as children, when they became older the growth stopped, and they matured to become smaller-than-normal adults, perhaps no taller than they would have been if they had never taken the hormone treatments.

The researchers said there is an "uncertain" possibility that the use of the hormone on healthy children may carry some adverse effects such as diabetes-like symptoms, overgrowth of breast tissue on men, carpal tunnel syndrome (a painful irritation of nerves in the hand and wrist) and possibly skin cancer.

The study called the practice of prescribing human growth hormone for normal but short children "a medical dilemma in which practical decisions about a therapy that aims to improve the quality of life must be made with promising but limited available data, absence of generally accepted guidelines and uncertainty about overall treatment effectiveness."

The researchers said using human growth hormone to make normally short children taller adds more than $120 billion to the national medical bill. The hormone treatment costs between $13,000 to $16,000 a year for each child, and they said the bill would be about $182 million if the treatment was used only for the 14,000 American children that suffer from the hormone deficiency.

Congress and the Food and Drug Administration investigated allegations two years ago that the two major U.S. manufacturers of human growth hormone, Eli Lilly & Co., of Indianapolis, and Genentech Inc., of San Francisco, were overpromoting their products. These probes ended, and no actions were taken. In addition, a Genentech vice president and three officials from the firm's distributor were cleared last year of federal charges alleging illegal kickbacks.

"From our company standpoint, we don't promote anything" outside of FDA prescribed uses, said Genentech spokeswoman Laura Leber. She noted that "it's up to the physician" how the hormone is used.

Nonetheless, the Case Western University researchers said using growth hormone for otherwise normal children raises questions about prescribing medicines to change the way people feel about themselves rather than to treat illness.