Managing Hereditary Angioedema in a Three-Generation Family: Danazol's Promise in Resource-Limited Settings

Background Hereditary angioedema (HAE) is a rare disorder in India, and while prevalence data is limited, it is believed that a significant number of individuals may be affected. Due to restricted access to first-line treatments, older therapies like danazol are commonly used despite associated risks in resource-constrained settings. This study aimed to assess the efficacy of danazol as an affordable long-term prophylaxis (LTP) for HAE in a three-generation family. Methods In this retrospective study, we analyzed demographic and clinical data from 22 HAE patients within a three-generation family, assessing serum C4 and C1 esterase inhibitor levels to confirm diagnosis. Patients were treated with danazol for LTP, and the effectiveness of treatment was evaluated using the angioedema control test (AECT), alongside monitoring for adverse effects. A paired-sample t-test was used to compare AECT scores before and after treatment. Results The participants had a mean age of 33.5 years and a male predominance (63.63%). The mean age of symptom onset was 20.4 years, and six died due to laryngeal edema episodes. Nearly all patients exhibited low serum C1-INH levels, confirming the diagnosis. Among the nine patients treated with danazol for LTP, AECT scores significantly improved from a mean of 7.9 to 9.8 (p = 0.003), with 90% experiencing reduced attack frequency. Mild adverse effects, such as weight gain and menstrual irregularities, were observed in 44.4% of treated patients. Conclusions This retrospective study demonstrated that danazol reduced attack frequency and severity in HAE patients in a resource-limited setting, with mild adverse effects observed in some individuals. While these findings support danazol as a cost-effective option for managing HAE, the results should be interpreted with caution due to the study's design limitations. The AECT proves to be a useful tool for evaluating the control of angioedema attacks. Because the risk of adverse effects is high, close monitoring of patients is mandatory. However, many patients accept the adverse effects of prophylactic treatment to avoid the distressing and sometimes life-threatening attacks of this condition. Regular monitoring remains essential to mitigate adverse effects, and prospective clinical trials are necessary to further validate the efficacy and safety of danazol for LTP.