Pharmacological manipulation of height: qualitative review of study populations and designs

Objective: Various substances that can have an important effect on height a re increasingly available. However, research into pharmacological manipulat ion of height in children has been criticized. There are concerns about dia gnostic criteria; about the medical, ethical, and economic ramifications of modulating growth in children with no endocrinological abnormalities; and about biased results due to weak study designs. The authors reviewed articl es published since Jan. 1, 1995, to characterize recent research into this area. Methods: 70 peer-reviewed articles published in 18 journals in 1995 describ ing effects of hormonal interventions to affect height were reviewed. Study population, intervention, main purpose (safety, physiology, or therapeutic effect), and methodology were examined. The search was expanded after 1995 to list randomized controlled trials (RCTs) investigating pharmacological manipulation in children and its effect on ultimate height in adults. Results: The inexpensive and brief androgen therapy for pubertal delay has been examined in RCTs, but expensive, long-term treatments to alter final a dult height in children have rarely been subjected to RCTs. Some outcome re ports pooled subjects with different causes of short stature. Documentation of growth hormone deficiency is problematic. Conclusions: There is a lack of RCTs in which target populations and growth outcomes are explicitly defined. Further research into overcoming barriers to relevant RCT studies is needed.