TREATMENT OF HEREDITARY ANGIOEDEMA WITH DANAZOL - REVERSAL OF CLINICAL AND BIOCHEMICAL ABNORMALITIES
TREATMENT OF HEREDITARY ANGIOEDEMA WITH DANAZOL - REVERSAL OF CLINICAL AND BIOCHEMICAL ABNORMALITIES
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DOI：
10.1056/nejm197612232952602
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发表时间：
1976-01-01
影响因子：
158.5
通讯作者：
FRANK, MM
中科院分区：
文献类型：
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作者：
GELFAND, JA;SHERINS, RJ;FRANK, MM
达那唑是一种雄激素衍生物，在一项双盲研究中对9例患者进行了预防遗传性血管性水肿发作的有效性评价。在47个安慰剂疗程中，44个以发作结束，但在46个达那唑疗程中仅发生1次发作。副作用很小，在研究的妇女中没有观察到男性化。C1 [补体第一组分]酯酶抑制剂水平增加3-4倍，补体第四组分（C4）水平增加15倍。这些变化开始于治疗的第1天，并在1-2周达到最大。停止治疗后，C1酯酶抑制剂和C4水平迅速下降。达那唑有效地防止遗传性血管性水肿的发作，并纠正潜在的生化异常。
Danazol, an androgen derivative, was evaluated for its effectiveness in preventing attacks of hereditary angioedema in a double-blind study with 9 patients. Of 47 placebo courses, 44 ended with attacks, but during 46 danazol courses only 1 attack occurred. Side effects were minimal, and virilization was not observed in the women studied. C1 [1st component of complement] esterase inhibitor levels increased 3-4 times and levels of the 4th component of complement (C4) increased 15 times. These changes began during the 1st day of therapy and were maximal by 1-2 wk. After therapy was stopped, C1 esterase inhibitor and C4 levels rapidly decreased. Danazol effectively prevents attacks in hereditary angioedema and acts to correct the underlying biochemical abnormality.