Novel treatment approaches: sex steroid blockade in children

新的治疗方法：儿童性类固醇阻断

• 批准号: 10916862
• 负责人: Deborah Merke
• 金额: --
• 依托单位: CLINICAL CENTER
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10916862
• 关键词: Acetates; Adult; Age; Androgen Antagonists; Androgens; Androstenedione; Aromatase Inhibitors; CYP17A1 gene; Child; Child Development; Cytochromes; Dose; Drops; Enzymes; Estrogens; Flutamide; Goals; Gonadal Steroid Hormones; Growth; Growth and Development function; Height; Hormone use; Hydrocortisone; Letrozole; Lyase; Measurement; Mixed Function Oxygenases; Patients; Peripheral; Phase; Prostate Cancer therapy; Randomized; Regimen; Serum; Testosterone; Treatment Protocols; United States National Institutes of Health; abiraterone; bone; clinical center; conventional therapy; design; follow-up; improved; inhibitor; novel; phase 1 study; prevent; prospective

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.

与常规治疗相比，抗雄激素（氟他胺）、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）可使线性生长速率和骨成熟正常化。一项针对成人身高的抗雄激素（氟他胺）和芳香化酶抑制剂（来曲唑）以及减少氢化可的松剂量与常规治疗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去随访的患者返回NIH临床中心，以便获得成人身高测量。