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When it became possible to synthetically produce growth hormone in the mid 1980s, a debate arose concerning whether short children without growth hormone deficiency, ie, idiopathic short stature (ISS), would benefit from treatment. Growth hormone treatment for ISS is based on two assumptions, ie, that treatment will increase final height and that short stature leads to psychological suffering. Treatment is administered by daily injections and may often continue for about 10 years. Currently, ISS is not an approved indication for growth hormone treatment. The number of children seeking treatment for ISS in Sweden is estimated at 500 to 1 000 per year.
In six controlled and two non-controlled studies, children with ISS received growth hormone treatment and were followed until they reached final height. The average effect of treatment on final height varies substantially among the studies, ranging from 2 cm to 9 cm. The distribution within the group was wide. Compiling the results of the studies suggests an average growth gain of 3 cm to 7 cm. It has not been established that growth hormone treatment reduces behavioral disorders and problems at school during childhood, or that treatment leads to a higher quality of life in adulthood. The rate of side effects from growth hormone treatment in children is low. Known, but uncommon, side effects include edema and diabetes.
Two important factors have ethical implications. First, one may question whether it is ethically defensible to subject healthy children to daily treatment for an extended period, with uncertain individual benefits. Second, the issue arises as to whether short stature in otherwise healthy individuals should be classified as a disease, or whether treatment that promotes growth in this group should be classified as "cosmetic". Hence, whether growth hormone treatment of children with ISS should be considered to be a task for health care should therefore be the subject of further discussions on prioritizations of medical needs versus resource allocation.
The cost-effectiveness of growth hormone treatment in ISS has been estimated in a meta-analysis and in an economic evaluation from the NHS R&D HTA Programme. The incremental cost of growth hormone treatment for one child was estimated at 650 000 to 900 000 SEK. The incremental cost per centimetre gained in final height was between 175 000 and 350 000 SEK.
There is moderate* scientific documentation concerning the patient benefits derived from treatment of idiopathic short stature. There is poor* scientific documentation on the cost-effectiveness of the method.
*This assessment by SBU Alert uses a 4-point scale to grade the quality and evidence of the scientific documentation. The grades indicate: (1) good, (2) moderate, (3) poor, or (4) no scientific evidence on the subject.
This summary is based on a report prepared at SBU in collaboration with Martin Ritzén, Professor Emeritus, Pediatric Endocrinology Laboratory, Astrid Lindgren’s Children’s Hospital at Karolinska Hospital, Stockholm, and Frida Meyer, Resident Physician, Mälar Hospital, Eskilstuna (Supervisor: Mona Britton, SBU). It has been reviewed by Kerstin Albertsson-Wikland, Professor, H M Queen Silvia Pediatric Hospital, Göteborg.
Alert is a joint effort by the Swedish Council on Technology Assessment in Health Care (SBU), the Medical Products Agency, the National Board of Health and Welfare, and the Federation of Swedish County Councils.
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