Long-Term Prophylaxis With Androgens in the Management of Hereditary Angioedema in Emerging Countries
November 2022
in “
Orphanet Journal of Rare Diseases
”
TLDR Long-term use of androgens can help manage Hereditary Angioedema (HAE) but may cause serious side effects, so alternative treatments with fewer side effects are being considered.
Hereditary Angioedema (HAE) is a rare genetic disease often managed with androgens, particularly in emerging countries like China and Brazil. Androgens, such as danazol, can reduce the frequency and severity of HAE attacks by increasing C1-INH mRNA expression and promoting accelerated degradation of bradykinin. However, long-term androgen treatment can lead to significant adverse effects, including virilization, weight gain, acne, liver damage, and anxiety/depression. In a survey of 107 Chinese HAE patients, 74.6% experienced significant adverse effects from androgen use. Alternative treatments, including plasma-derived C1 inhibitor, recombinant human C1 inhibitor, plasma Kallikrein inhibitor, and bradykinin B2 receptor antagonist, have shown satisfactory results with mostly non-serious side effects, potentially changing the standard of HAE management.