Growth hormone deficiency medical therapy
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]
Overview
Medical Therapy
GH deficiency is treated by replacing GH. Until 1985, growth hormone for treatment was obtained by extraction from human pituitary glands collected at autopsy. Since 1985 GH is a synthetic copy of human GH, manufactured by recombinant DNA technology.
Treatment of GH deficiency in childhood
When treated with GH, a severely deficient child will begin to grow faster within months. In the first year of treatment, the rate of growth may increase from half as fast as other children are growing to twice as fast (e.g., from 1 inch a year to 4 inches, or 2.5 cm to 10). Growth typically slows in subsequent years, but usually remains above normal so that over several years a child who had fallen far behind in his height may grow into the normal height range. Parents often notice increased strength, appetite, and energy. Increased muscle strength may allow young children to overcome delays of motor development. Excess adipose tissue may be reduced.
There are almost no significant side effects of this type of physiologic replacement. Rare risks and unsettled issues are discussed in the article on GH treatment, but GH deficient children receiving replacement doses are at the lowest risk for problems and receive the greatest benefit.
Nevertheless, costs of treatment in terms of money, effort, and perhaps quality of life, are substantial. Treatment usually involves daily injections of growth hormone for children. Most pediatric endocrinologists monitor growth and adjust dose every 3-4 months and many of these visits involve blood tests. Treatment is usually extended as long as the child is growing, and lifelong continuation may be recommended for those most severely deficient. Nearly painless insulin syringes and pen injectors reduce the discomfort. Most children and families are enthusiastic once the benefits begin to be seen. Treatment is expensive - as much as $US 10,000 to 30,000 a year is common.
Little except the cost of treating severely deficient children is controversial, and it is likely that the majority of children with severe growth hormone deficiency in North America, Japan, and much of Europe and the rest of the developed world are offered treatment, and most accept. The story is very different for adult deficiency.
It has been shown repeatedly in research studies that GH treatment can confer a number of measurable benefits to severely GH-deficient adults, such as enhanced energy and strength, and improved bone density. Muscle mass may increase at the expense of adipose tissue. Blood lipid levels improve, but long term mortality benefit has not yet been demonstrated.
GH for severe adult deficiency is usually prescribed as three injections per week at a weekly dose about 25% of children's doses and comparably lower cost. Despite the demonstrated benefits, most adults with GH deficiency are not being treated due to a combination of factors such as unwillingness of young adults to seek medical care, unacceptability of injections, inadequate insurance coverage, and significantly lower rates of diagnosis and treatment offer by internist endocrinologists.