Growth hormone deficiency: optimizing therapy and new issues

Growth Hormone Deficiency (GHD) with low circulating IGF1 requires replacement therapy. Paradoxically, it remains a controversial issue in a large part of patients, those considered as having isolated GHD of the idiopathic milder form. Challenges remain in this area in spite of intensive and sometimes controversial studies. This is true for the diagnosis of the milder forms (also called partial GHD), for the assessment of the growth response and the evaluation of final height benefit. In addition the cost-benefit issue should not be ignored. Therefore, the author tried to review data relevant to the evaluation of GH secretion which even now remains largely arbitrary. The growth response, which is the primary therapeutic goal in these children should also be carefully discussed as reported in recent papers. Focusing on individual responses should help adjusting individual dosage within the standard recommended doses, but one should also remember that there are no long term safety data for non conventional high rhGH doses. More studies are needed. Response to treatment during the first year may in the future help select the patients who are prone to the benefit of long term rhGH therapy. Basic rules for indication and progression of treatment are proposed in children with various forms of GHD. It is also remarkable that the present safety data are all coming from several post-marketing studies. This means that long term independent studies are now required as recombinant growth hormone remains the most appropriate and efficient therapy when permanent GH deficiency is fully documented.