Should we treat genetic syndromes?

With the greater availability of GH due to the development of recombinant human GH, GH therapy has expanded well beyond the original FDA indication for GHD. We examine the NCGS database, comparing data for FDA-approved indications with data for 'other' conditions to see whether such expanded use is warranted. Although statistical analyses are of questionable validity because of the small size of some subpopulations and other factors discussed herein, certain trends emerge from the data captured by NCGS. We conclude that many clinical syndromes characterized by short stature are responsive to GH, at least in the short term. For many such syndromes, responsiveness is of the same magnitude as that seen in Turner's syndrome and, occasionally, GHD. If responsiveness to GH is the most important criterion for GH therapy, these 'other' conditions warrant an open-minded, prospective evaluation.