Measuring the benefits of growth hormone therapy in children: a role for preference-based approaches?

摘要

Recombinant human growth hormone has revolutionised the management of children with growth hormone deficiency, chronic renal insufficiency, Turner syndrome, Prader-Willi syndrome, small-forgestational-age status, idiopathic short stature and other growth disorders in recent decades. The benefits of administering growth hormone to children have commonly been measured in terms of acceleration in linear growth. Although informative, lines of enquiry that measure the benefits of recombinant human growth hormone in natural or physical units, such as acceleration in linear growth, suffer from a number of limitations. Most notably, they are unable to incorporate the several health changes that can result from growth hormone therapy, including the increased risk of a number of adverse effects such as pigmentation and growth of nevi, gynecomastia, pancreatitis and benign intracranial hypertension, within a single measure. In addition, they overlook the broader effects that the treatment may have on children's physical, mental and social well-being, as well individual preferences for those health changes.