Is testosterone therapy used to treat pediatric short stature?

Testosterone is Not Recommended for Pediatric Short Stature

Growth hormone (GH) therapy, not testosterone, is the recommended treatment for pediatric short stature in specific clinical scenarios, as testosterone can accelerate bone maturation without compensatory linear growth, potentially compromising final adult height. 1

Appropriate Treatments for Pediatric Short Stature

Growth Hormone Therapy Indications

GH therapy is indicated in the following conditions:

• Children with chronic kidney disease (CKD) stages 3-5 or on dialysis when:

  • Height is below the 3rd percentile AND
  • Height velocity is below the 25th percentile for at least 3 months in infants or 6 months in children 2, 3
  • Other treatable factors (nutrition, metabolic acidosis, renal osteodystrophy) have been addressed

• Children with genetic conditions:

  • SHOX gene-related disorders
  • Turner syndrome
  • Other specific genetic causes of short stature 3, 4

• Children with idiopathic short stature (ISS):

  • Height below -2.25 SDS in the United States and seven other countries 4
  • Expected increase in adult height: 3.5-7.5 cm 4

Why Testosterone is Contraindicated

Testosterone and other anabolic steroids are not recommended for pediatric short stature because:

1. They cause disproportionately rapid epiphyseal maturation that can compromise final adult height 1, 5
2. They produce rapid masculinization, which is undesirable in most pediatric cases 5
3. The FDA specifically warns that in children, "androgen treatment may accelerate bone maturation without producing compensatory gain in linear growth" 1
4. This adverse effect can result in compromised adult stature, with younger children facing greater risk 1

Diagnostic Evaluation Before Treatment

Before initiating any treatment for short stature:

• Define short stature as height-for-age less than 2 standard deviations below the mean 3
• Conduct comprehensive evaluation including:
  • Genetic testing (chromosomal analysis, especially for girls to rule out Turner syndrome) 3, 6
  • Thyroid function tests 3
  • Assessment for skeletal dysplasia in children with disproportionate short stature 3
  • Bone age assessment of wrist and hand 2

Monitoring During GH Treatment

For children receiving GH therapy:

• Monitor bone age every 6 months 2, 3
• Monitor IGF-1 levels for safety and dosage adjustment 3
• Perform fundoscopic examination before starting therapy to rule out papilledema 3
• Assess first-year response (successful if height SDS increases >0.3-0.5) 3, 4
• Continue until epiphyseal fusion occurs 3

Pitfalls and Caveats

• Approximately 50% of patients referred for evaluation have constitutional delay of growth or familial short stature, which are non-pathological conditions 3
• Individual responses to GH therapy vary significantly 3, 4
• GH therapy is contraindicated in active malignancy, uncontrolled diabetes mellitus, pre-existing papilledema, and known hypersensitivity to growth hormone 3
• The younger the child, the greater the risk of compromising final mature height with androgen therapy 1
• Oxandrolone (a specific anabolic steroid) may have a more favorable anabolic/androgenic ratio than testosterone for growth promotion in specific cases, but GH remains the first-line therapy 5

In conclusion, while testosterone has been historically used for short stature, current evidence strongly supports GH as the appropriate treatment for pediatric short stature in specific conditions, with testosterone being contraindicated due to its adverse effects on bone maturation and final adult height.