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使用氢化可的松治疗 2 1羟化酶缺陷的先天性肾上腺皮质增生症 (CAH)已经 5 0余年 ,但雄激素过多所致的多毛症、生长发育异常、不育症、医源性Cushing综合征等并发症则较常见。本文介绍了一些新的临床试验方案和一些有前途的前临床研究 :减少氢化可的松的剂量 ,调整服药时间及选择新的剂型 ,使用抗雄激素药物和芳香化酶抑制剂、促黄体生成素释放激素类似物、生长激素、促肾上腺皮质激素释放激素拮抗剂、基因治疗等。这些疗法均有望提高CAH病人的生活质量 ,但仍需作进一步观察
Corticosteroid deficiency (CAH) has been treated with hydrocortisone for more than 50 years, but hirsutism caused by hyperandrogenism, growth and development abnormalities, infertility, iatrogenic Cushing Syndromes and other complications are more common. This article describes a number of new clinical trial protocols and promising preclinical studies that reduce the dose of hydrocortisone, adjust the duration of administration and select new dosage forms, use anti-androgens and aromatase inhibitors, and promote luteinizing Hormone releasing hormone analogs, growth hormones, corticotropin releasing hormone antagonists, gene therapy and the like. These treatments are expected to improve the quality of life of patients with CAH, but still need further observation