Novel treatment approaches: sex steroid blockade in children

研究表明，与常规治疗相比，使用抗雄激素(氟他胺)、芳香化酶抑制剂(来曲唑)和减少氢化可的松剂量的研究方案在短期(2年)内使线性生长速度和骨成熟正常化。一项关于成人身高的前瞻性随机平行研究即将完成，该研究采用抗雄激素(氟他胺)和芳香酶抑制剂(来曲唑)，并减少氢化可的松剂量与常规治疗。现在正在联系辍学或失去随访的患者返回NIH临床中心，以便获得成人身高测量。 在另一项研究中，阿比拉特龙是17羟基酶/C17，20裂解酶(细胞色素P450c17 CYP17)的不可逆抑制剂，是合成睾酮所需的关键酶，正在进行一项针对青春期前儿童的多中心1/2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.