Lournaris Torres-Santiago, Nelly Mauras

Approach to the Peripubertal Patient with Short Stature

  • Biochemistry (medical)
  • Clinical Biochemistry
  • Endocrinology
  • Biochemistry
  • Endocrinology, Diabetes and Metabolism

Abstract Context The assessment and treatment of growth-retarded children is increasingly complex, and due to availability of targeted genetic sequencing, an ever-expanding list of conditions are being identified causing growth retardation. Among endocrine-related etiologies of short stature amenable to hormonal treatment, defects in the GH-IGF-I axis remain pre-eminent, with a multiplicity of disorders causing decreased secretion or insensitivity to GH action. Sex steroids in puberty increase epiphyseal senescence and eventual growth plate closure. This is mediated mostly via estrogen receptor (ER)α in males and females, effects that can greatly limit time available for growth. Evidence Acquisition Extensive literature review through PubMed and other search engines. Evidence Synthesis Therapeutic strategies to be considered in peripubertal and pubertal children with disordered growth are here discussed, including daily and weekly GH, low-dose sex steroids, GnRH analogues in combination with GH, aromatase inhibitors (AIs) alone and in combination with GH in boys. When used for at least 2-3 years GnRH analogues combined with GH can result in meaningful increases in height. AIs used with GH permit puberty to progress in boys without hindrance, allowing for greater selectivity in only decreasing the estrogen, also resulting in taller height. With more than 20 years of cumulative experience in the use of these medications clinically, the safety profile of these treatments is also discussed. Conclusions The approach of growth retardation in the peripubertal and pubertal years must consider the sex steroid milieu and the tempo of bone acceleration. Treatment of affected children in this period must be individualized.

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