Long-Term Follow Up and Treatment Outcomes of A 2 Year-Old-Boy With Metastatic Testosterone-Secreting Adrenocortical Carcinoma

ABSTRACT Objective Treatment of metastatic adrenocortical carcinoma (ACC) is challenging and long-term survival rates are exceedingly low. Long-term outcome data for pediatric patients who received mitotane is very limited. Methods We describe the case of a 2-year-old boy with ACC with a lung metastasis. He was treated with surgery, chemotherapy, and mitotane, and remains disease-free 13 years after diagnosis. Results The key endocrine issues learned from this case include: adrenal-derived sex-steroid and insulin-like growth factor-2 levels are correlated with disease status; very high doses of glucocorticoid and mineralocorticoid are required while on treatment of mitotane; and central precocious puberty needs to be detected and treated in a timely manner to preserve final adult height. Conclusion We report a case of pediatric ACC with metastasis that was successfully treated with surgery, chemotherapy, and adjuvant therapy with mitotane. Appropriate endocrine testing and management are important for long-term survival and quality of life. Abbreviations ACC adrenocortical carcinoma IGF insulin-like growth factor IGFBP insulin-like growth factor binding protein