Near-final height outcomes in children with idiopathic short stature responsive to the IGF-1 generation test: a multicenter retrospective study of rhGH therapy.

rhGH therapy was associated with improvements in height SDS and near-final height in this selected cohort of children with idiopathic short stature.

However, the absence of an untreated control group and the minimal difference between near-final height and baseline predicted adult height indicate that these findings should be interpreted with caution.

Although no GH1 mutations were identified, the relatively high rate of parental consanguinity suggests that other genetic factors within the GH-IGF-1 axis or epiphyseal growth plate pathways may underlie the etiology of short stature.