Novel approaches to short stature therapy

Best Pract Res Clin Endocrinol Metab. 2015 Jun;29(3):353-66. doi: 10.1016/j.beem.2015.01.003. Epub 2015 Feb 7.


Besides growth hormone, several pharmaceutical products have been investigated for efficacy and safety in increasing short term growth or adult height. Short-term treatment with testosterone esters in boys with constitutional delay of growth and puberty is efficacious in generating secondary sex characteristics and growth acceleration. The addition of oxandrolone to growth hormone (GH) in Turner syndrome has an additive effect on adult height gain. Treatment with GnRH analogs is the established treatment of central precocious puberty, and its addition to GH therapy appears effective in increasing adult height in GH deficient children, and possibly short children born SGA or with SHOX deficiency, who are still short at pubertal onset. Aromatase inhibitors appear effective in several rare disorders, but their value in increasing adult height in early pubertal boys with GH deficiency or idiopathic short stature is uncertain. A trial with a C-natriuretic peptide analog offers hope for children with achondroplasia.

Keywords: achondroplasia; aromatase inhibitors; delayed puberty; gonadotropin-releasing hormone analogs; growth hormone; oxandrolone; precocious puberty; short stature.

Publication types

  • Review

MeSH terms

  • Achondroplasia / drug therapy
  • Androgens / therapeutic use*
  • Aromatase Inhibitors / therapeutic use*
  • Dwarfism, Pituitary / drug therapy
  • Gonadotropin-Releasing Hormone / analogs & derivatives
  • Growth Disorders / drug therapy*
  • Human Growth Hormone / therapeutic use*
  • Humans
  • Natriuretic Agents / therapeutic use*
  • Natriuretic Peptide, C-Type / therapeutic use*
  • Oxandrolone / therapeutic use
  • Puberty, Precocious / drug therapy
  • Recombinant Proteins
  • Testosterone / therapeutic use
  • Turner Syndrome / drug therapy


  • Androgens
  • Aromatase Inhibitors
  • Natriuretic Agents
  • Recombinant Proteins
  • Human Growth Hormone
  • Natriuretic Peptide, C-Type
  • Gonadotropin-Releasing Hormone
  • Testosterone
  • Oxandrolone