a floor effect. Consequently, new and more efficient measures need to be tested and developed.
Investigators may wish to consider several strategies in developing new measures to assess QOL in children and adolescents with short stature. One initial strategy is to identify key target domains that have previously been shown to be clinically relevant and responsive to change in adult populations. In-depth interviews of children of short stature with and without proven GH insufficiency, as well as interviews with their parents and siblings, would be useful in developing new measures. Moreover, it would be useful to obtain information concerning the benefits that clinicians observe after institution of GH therapy in order to design questions that are especially sensitive to these changes.
Future research should utilize prospective designs and identify the differences between truly GH-insufficient children versus those with a modest degree of insufficiency. Using general as well as specific measures, investigators can identify the impact of GH insufficiency and short stature on general and stature-related QOL. If possible, investigators can use crossover designs to optimize the treatment comparison and reduce the sample size that is necessary. Reanalyzing data on a single item level over time and across comparison groups may help identify potent discriminant items in QOL assessments. In evaluating children's response to GH insufficiency and short stature, investigators should look carefully at age differences. Differences in QOL may be more apparent in older versus younger age cohorts.
Finally, in evaluating treatments for GH insufficiency, investigators should consider measuring a range of possible outcomes at different time points. For example, improvement in the child's current functioning may not be the only definition of successful therapy. Assessment of a treatment's ability to prevent future problems in adolescents and adults with GH insufficiency is also an important goal that has yet to be realized.
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