目的评价达那唑治疗遗传性血管性水肿(HAE)的疗效和安全性。方法收集1985至2010年于北京协和医院就诊,应用达那唑≥1年、随访时间≥1年或随访次数≥5次并有完整随访记录的HAE患者的临床资料进行回顾性分析。疗效评估指标为用药前后皮肤水肿、腹痛、喉头水肿发作频率、血清补体第1成分抑制因子(C1INH)含量与功能、血清补体第4成分(C4)含量。安全性评价指标为用药前后肝功能、体重、女性患者月经情况变化。结果 24例患者符合纳入标准。男、女性各12例,就诊年龄(36±15)岁,病程(15±8)年。达那唑初始剂量均为200 mg,2~3次/d,口服1~4周逐渐减至维持剂量,男性维持剂量为(169±94)mg/d,女性为(130±56)mg/d。24例患者服用达那唑时间的中位数和四分位数间距为5(1.1~10.3)年。服用达那唑之前,24例患者体表水肿、喉头水肿和腹痛发生率分别为100%(24例)、70.8%(17例)和62.5%(15例),治疗后分别降至41.6%(10例)、12.5%(3例)和8.3%(2例),差异均有统计学意义(均P<0.01)。服用达那唑1~4周后,24例患者血清C1INH含量和功能分别由治疗前的(0.08±0.06)g/L和(0.14±0.04)U/ml升至(0.12±0.07)g/L(P=0.05)和(0.26±0.05)U/ml(P<0.01),但未恢复至正常水平。22例患者有血清C4含量检测结果记录,2004年前后就诊者分别为17和5例,服用达那唑前后C4含量分别为(23.5±12.6)、(56.9±30.0)mg/L和(0.06±0.01)、(0.08±0.01)g/L,差异均有统计学意义(均P<0.05)。2例患者分别在服药30和45 d后血清丙氨酸转氨酶由25和20 U/L升至138和74 U/L,并出现脱发、油脂分泌增多、烦躁等症状,给予保肝治疗后恢复正常。6例体重较服药前增加。6例女性患者分别出现停经、月经周期延长或缩短、月经量减少或增多等症状,将达那唑剂量减至200 mg,1次/d或隔日1次后,月经紊乱得到明显改善。结论达那唑治疗HAE有效且安全。建议将血清C4水平作为调整达那唑剂量的指标。 Objective To evaluate the efficacy and safety of danazol in the treatment of hereditary angioedema (HAE). Methods The clinical data of HAE patients who were treated at Dancow Hospital of Peking Union Medical College from 1985 to 2010 and who were treated with danazol for more than one year, followed up for more than one year or followed up for more than five times and with complete follow-up were retrospectively analyzed. The indexes of curative effect were skin edema, abdominal pain, frequency of laryngeal edema, content and function of C1INH and C4 in serum before and after treatment. Safety evaluation index before and after treatment of liver function, body weight, changes in women’s menstrual conditions. Results 24 patients met the inclusion criteria. 12 males and 12 females, with a mean age of 36 ± 15 years and a duration of 15 ± 8 years. The initial dose of danazol was 200 mg, 2 to 3 times / d, and gradually decreased to the maintenance dose from 1 to 4 weeks after oral administration. The maintenance dose of danazol was (169 ± 94) mg / d in women and (130 ± 56) mg / d. The median and quartile spacing of 24 patients with danazol was 5 (1.1 to 10.3) years. Before taking danazol, the incidences of surface edema, laryngeal edema and abdominal pain were 100% (24 cases), 70.8% (17 cases) and 62.5% (15 cases) in the 24 patients, respectively, and were reduced to 41.6% 10 cases), 12.5% ​​(3 cases) and 8.3% (2 cases). The differences were statistically significant (all P <0.01). After 1 to 4 weeks of danazol treatment, the level of serum C1INH and the function of 24 patients increased from (0.08 ± 0.06) g / L and (0.14 ± 0.04) U / ml to (0.12 ± 0.07) g / L (P = 0.05) and (0.26 ± 0.05) U / ml (P <0.01), but did not recover to normal level. The results of serum C4 levels were recorded in 22 patients. Before and after 2004, there were 17 and 5 patients respectively. Before and after taking danazol, C4 contents were (23.5 ± 12.6), (56.9 ± 30.0) mg / L and (0.06 ± 0.01), (0.08 ± 0.01) g / L, the difference was statistically significant (all P <0.05). Serum alanine aminotransferase increased from 25 and 20 U / L to 138 and 74 U / L in 2 patients after 30 and 45 d administration, respectively. The symptoms of alopecia, such as alopecia, normal. 6 cases of body weight increased compared with before treatment. 6 cases of female patients were menopause, menstrual cycle to extend or shorten, reduce or increase menstrual symptoms such as danazol dose to 200 mg, 1 times / d or every other day, menstrual disorders were significantly improved. Conclusion Danazol is effective and safe in treating HAE. It is recommended that serum C4 levels be used as an indicator of dose adjustment for danazol.