Arlan L. Rosenbloom, MD

Promotion of off-label use of recombinant human insulin-like growth factor I began as soon as FDA orphan drug approval was given late in 2005 for its use in growth hormone insensitivity due to growth hormone receptor deficiency or growth hormone inactivating antibodies following recombinant human growth hormone treatment of children with growth hormone gene deletion. This promotion was based on the hypothesis that milder forms of growth hormone insensitivity accounted for a substantial proportion of unexplained short stature.

Some 2% of children will always be more than two standard deviation below the height mean for age and sex. A more appropriate definition of short stature for clinical purposes would be 2 SD below the mean parental standard deviation score. Because idiopathic short stature is a diagnosis of exclusion, it is necessary to clarify what it is not.