Hormonal Treatment of Idiopathic Short Stature

Abstract

Background and Objective: The classification idiopathic short stature (ISS) represents a heterogeneous group of children who, as a group, are similar in height to patients with growth hormone (GH) deficiency, Turner syndrome, and short stature as a consequence of being born small for gestational age. GH therapy has been an effective treatment for enhancing height in children with ISS. Methods: A large body of literature – including data from KIGS (Pfizer International Growth Study Database) and the National Cooperative Growth Study database – shows that non-placebo-controlled GH treatment programs generally, though not uniformly, are associated with substantial increments in height gain. Placebo-controlled trials conducted in several countries report significant gains of 5 to 8 cm in final height. Conclusions: Long-term data are needed to assess the behavioral consequences, if any, of GH treatment for children with ISS. As the pool of children with ISS is potentially large, it would be highly desirable if clinical trials enabled accurate prediction of likely height gain and could result in greater individualization of treatment. The use of aromatase inhibitors to slow estrogen-mediated skeletal maturation is a new treatment modality, but it has only been minimally tested. The lower cost and ease of oral administration are attractive considerations, but long-term safety data are needed.