Is testosterone therapy recommended for pediatric short stature?

Testosterone Therapy is Not Recommended for Pediatric Short Stature

Testosterone therapy is not recommended for pediatric short stature as it can accelerate bone maturation without compensatory linear growth, potentially compromising final adult height. 1, 2

Appropriate Management of Pediatric Short Stature

Growth Hormone (GH) Therapy - Primary Recommendation

For children with short stature, growth hormone therapy is the recommended treatment in specific conditions:

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

  • Older than 6 months of age
  • Height below 3rd percentile AND height velocity below 25th percentile
  • Persistent growth failure for at least 3 months (infants) or 6 months (children)
  • After addressing other treatable factors affecting growth 3, 4

• Children with genetic conditions associated with short stature:

  • SHOX gene-related disorders
  • Turner syndrome (requires chromosomal analysis in girls with short stature) 4

Contraindications and Risks of Testosterone Use

Testosterone therapy in pediatric patients with short stature carries significant risks:

• Premature bone maturation: Accelerates epiphyseal closure without compensatory linear growth gain 1, 2
• Compromised adult height: The younger the child, the greater the risk of reducing final adult height potential 2
• Hepatic complications: Risk of hepatic adenomas, hepatocellular carcinoma, and peliosis hepatis with prolonged use 1
• Cardiovascular risks: Edema with potential congestive heart failure in predisposed patients 2

Monitoring Requirements for Growth Hormone Therapy

If GH therapy is initiated:

• Bone age assessment every 6 months to monitor bone maturation
• IGF-1 level monitoring for safety and dosage adjustment
• Fundoscopic examination before starting therapy to rule out papilledema 4

Expected Outcomes with Growth Hormone Therapy

• First-year growth response: 10.8 cm/yr (compared to 6.5 cm/yr in controls) 4
• Successful response defined as increase in height SDS >0.3-0.5 in first year 4
• Mean increase in adult height: 3.5-7.5 cm after 4-7 years of treatment 5
• Individual responses vary significantly 3, 4

Special Considerations

Diagnostic Evaluation

• Short stature defined as height-for-age less than 2 standard deviations below mean (below 3rd percentile)
• Comprehensive genetic testing increasingly important for identifying specific causes 4, 6
• SHOX gene testing recommended for children with subtle skeletal changes or significant short stature (>3 SD) 4

Treatment Duration

• GH therapy should continue until epiphyseal fusion occurs 4
• Prioritize patients with most severe height deficits and documented conditions known to respond to GH therapy 4

Clinical Pitfalls to Avoid

1. Using testosterone to accelerate growth: This can permanently compromise adult height by prematurely closing growth plates 1, 2

2. Delaying treatment: Early intervention with appropriate therapy (GH when indicated) may be more cost-effective and yield better outcomes than waiting until growth retardation is severe 3

3. Misdiagnosis: Approximately 50% of patients referred for genetic evaluation of short stature have constitutional delay of growth or familial short stature, which are non-pathological conditions 4

4. Overlooking underlying conditions: Comprehensive evaluation is essential to identify specific causes of short stature that may respond to targeted therapies 6

The evidence clearly demonstrates that while growth hormone therapy has established benefits for specific conditions causing short stature, testosterone therapy should be avoided as it can compromise final adult height potential in pediatric patients with short stature.