Novel treatment approaches: sex steroid blockade in children

与常规治疗相比，抗雄激素（氟替卡松）、芳香酶抑制剂（来曲唑）和减少氢化可的松剂量的研究方案显示短期（2年）可使线性生长率和骨成熟正常化。一项关于抗雄激素药物（氟替卡松）和芳香酶抑制剂（来曲唑）以及减少氢化可的松剂量与常规治疗相比对成人身高的前瞻性长期随机平行研究正在完成。 现在正在联系退出或失访的患者返回NIH临床中心，以获得成人身高测量。 在一项单独的研究中，阿比特龙是一种17羟化酶/C17，20-裂解酶（细胞色素P450 c17 CYP 17）（睾酮合成所需的关键酶）的不可逆抑制剂，目前正在青春期前儿童中进行多中心I/II期研究。

The investigational regimen of an antiandrogen (flutamide), an aromatase inhibitor (letrozole), and reduced hydrocortisone dose vs. conventional treatment was shown short-term (2 year) to normalize linear growth rate and bone maturation. A prospective long-term randomized parallel study to adult height of an antiandrogen (flutamide) and an aromatase inhibitor (letrozole), and reduced hydrocortisone dose vs. conventional treatment is being completed. Patients that dropped out or were lost to follow-up are now being contacted to return to the NIH Clinical Center in order to obtain an adult height measurement. In a separate study, Abiraterone, an irreversible inhibitor of 17 hydroxylase/C17,20-lyase (cytochrome P450c17 CYP17), a key enzyme required for testosterone synthesis, is being studied in a multicenter Phase 1/2 study in pre-pubescent children.