Oxandrolone therapy in pediatric growth disorders: A comprehensive review of benefits, risks, and clinical implications

Background: Oxandrolone, a synthetic anabolic steroid, has been used in pediatric endocrinology and metabolic recovery for its growth-promoting and muscle-preserving properties. It has demonstrated efficacy in constitutional delay of growth and puberty (CDGP), Turner syndrome (TS), and severe burn recovery. However, concerns regarding skeletal maturation acceleration, virilization, and metabolic effects necessitate a comprehensive evaluation of its benefits and risks.

Objective: This review aims to assess the therapeutic effects of oxandrolone in pediatric growth disorders, analyzing height velocity improvements, final adult height outcomes, metabolic benefits, and potential adverse effects. The objective is to provide an evidence-based framework to guide clinical decision-making for its safe and effective use in children and adolescents.

Methods: A systematic evaluation of 45 peer-reviewed studies published between 2000 and 2024 was conducted, encompassing randomized controlled trials (RCTs), observational studies, and meta-analyses. Studies included pediatric patients (ages 2–18 years) receiving oxandrolone for CDGP, TS, or burn recovery. Data extraction focused on growth velocity, final height, lean body mass, metabolic outcomes, and adverse effects. Statistical analyses included mean differences (MDs), standardized mean differences (SMDs), and odds ratios (ORs) to compare treatment efficacy and safety outcomes.

Results

• Growth Promotion: Oxandrolone therapy increased growth velocity by 4.5–9.6 cm/year in CDGP and TS patients, with an additional 2.3–4.6 cm in final adult height when used with GH therapy.

• Metabolic Benefits: In pediatric burn recovery, oxandrolone enhanced lean body mass retention (+12.3%), improved bone mineral content, and accelerated wound healing.

• Safety and Adverse Effects: Virilization (12.5%–15.2%), skeletal maturation acceleration (3–7 months), and mild lipid profile alterations (10%–12%) were observed in Turner syndrome patients, with higher risks at doses >0.06 mg/kg/day. No severe adverse effects were reported in burn recovery patients.

Conclusion: Oxandrolone therapy substantially benefits pediatric growth promotion and metabolic recovery, particularly in CDGP, Turner syndrome, and burn injuries. However, dose-dependent risks such as virilization, lipid alterations, and accelerated skeletal maturation necessitate careful monitoring. Combining GH with oxandrolone enhances efficacy, and patient selection criteria must be optimized to balance risks and benefits. Future research should focus on long-term metabolic effects, personalized dosing strategies, and safer alternative therapies to further refine its clinical applications in pediatric endocrinology.