Cicardi M, Bergamaschini L, Cugno M, Hack E, Agostoni G, Agostoni A
Clinica Medica, Ospedale S. Paolo, Università di Milano, Italy.
J Allergy Clin Immunol. 1991 Apr;87(4):768-73. doi: 10.1016/0091-6749(91)90120-d.
Fifty-six patients affected with hereditary angioedema have been followed during long-term prophylaxis with attenuated androgens. The treatment was started in patients who had one or more severe attacks per month. In 24 patients, the therapy lasted for more than 5 years. The minimal effective dose usually did not exceed 2 mg/day of stanozolol or 200 mg/day of danazol. Only in two patients were these doses not sufficient to achieve the complete disappearance of symptoms. Irregular menstruation, but rarely amenorrhea, was the only significant side effect. One patient had to stop the therapy because of laboratory signs of hepatic cell necrosis. In one patient, danazol was administered during the last 8 weeks of pregnancy without side effects for the mother but with transient signs of virilization for the female baby. To find a biochemical marker for the minimal effective dose of androgen derivatives, we measured the plasma levels of C1 C1 INH complexes at different doses of stanozolol in four patients with hereditary angioedema. We found that these complexes, elevated before treatment, promptly reverted to normal values during androgen therapy and remained normal with any reduction of the dose of the drug as long as the patient remained symptom free. Therefore, the measurement of C1 C1 INH complexes appears to reflect the activity of the disease and not the amount of androgen that is administered.
56例遗传性血管性水肿患者在接受减毒雄激素长期预防治疗期间接受了随访。治疗开始于每月发作一次或多次严重发作的患者。24例患者的治疗持续了5年以上。最小有效剂量通常不超过司坦唑醇2mg/天或达那唑200mg/天。只有2例患者这些剂量不足以使症状完全消失。月经不调是唯一显著的副作用，但闭经很少见。1例患者因肝细胞坏死的实验室指标而不得不停止治疗。1例患者在妊娠最后8周使用达那唑，对母亲无副作用，但女婴有短暂的男性化体征。为了找到雄激素衍生物最小有效剂量的生化标志物，我们在4例遗传性血管性水肿患者中测量了不同剂量司坦唑醇下血浆C1 C1 INH复合物的水平。我们发现，这些复合物在治疗前升高，在雄激素治疗期间迅速恢复到正常水平，并且只要患者无症状，随着药物剂量的任何减少仍保持正常。因此，C1 C1 INH复合物的测量似乎反映了疾病的活性，而不是所给予雄激素的量。
