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Prophylaxis of attacks of hereditary angioedema
Insights
Danazol effectively prevents hereditary angioedema attacks by increasing C1 INH levels. This study determined the minimum effective dose for prophylaxis, finding it ranges from 100-400 mg/day.
Area of Science:
- Immunology
- Pharmacology
Background:
- Hereditary angioedema (HAE) is characterized by C1 inhibitor (C1 INH) and C4 deficiency.
- Danazol, an attenuated androgen, is a potential prophylactic treatment for HAE.
Observation:
- Four HAE patients with monthly attacks and low C1 INH/C4 levels participated.
- The study aimed to identify the minimum effective Danazol dose for HAE prophylaxis.
Findings:
- Danazol therapy significantly reduced HAE attacks (6 attacks over 60 patient-months).
- The minimum effective dose ranged from 100 to 400 mg/day.
- Danazol increased serum C1 INH levels, normalizing them in 50% of patients.
Implications:
- Danazol demonstrates efficacy in preventing hereditary angioedema attacks.
- Treatment requires individualized dosing based on patient response.
- Further research into C1 INH modulation by androgens is warranted.
Abstract:
Danazol, an attentuated androgen, has been suggested as an effective agent for the prophylaxis of attacks of hereditary angioedema. Four patients, with a clinical history of hereditary angioedema and a demonstrated depression of the serum inhibitor of the first component of complement (C1 INH) and the fourth component of complement (C4), were entered into a study to determine the minimum effective dose of this agent. All four of the patients had been experiencing attacks at least monthly, but they had only six attacks during a total of 60 patient months of Danazol therapy. The minimum effective dose varied from 100 to 400 mg/day. The drug appeared to work by increasing the level of serum C1 INH which reached the normal range in two of four patients. Side effects were only the anticipated menstrual irregularities in the female patients. Danazol appears to be an efficacious drug for prophylaxis of hereditary angioedema.