The safety and effectiveness of human growth hormone using pharmacological dosing.

Human growth hormone in currently recommended dosage is effective in many short children, irrespective of their endogenous growth-hormone status. This suggests that present dosing is pharmacological rather than physiological. As for any drug, issues of safety should be of paramount concern. Reassuring short-term data with pharmacological dosing or long-term data with replacement dosing cannot guarantee the ultimate safety of this form of therapy. The risk of future malignancy should be of particular concern. Poorly growing children without classic (severe) growth-hormone deficiency constitute an increasing proportion of children treated with human growth hormone. There are no satisfactory criteria for the diagnosis of neurosecretory growth-hormone dysfunction. The closer to puberty these children are treated, the less likely it is that there will be benefits in terms of increased final height. Recommendations as to a 'safety first' approach to growth-hormone treatment are given. A multicentre controlled trial is urgently needed to establish the benefits of treating children with neurosecretory growth hormone dysfunction.