Short‐term prophylaxis in hereditary angioedema due to deficiency of the <scp>C</scp>1‐inhibitor – a long‐term survey

<jats:title>Abstract</jats:title><jats:sec><jats:title>Background</jats:title><jats:p>Hereditary angioedema is a potentially life‐threatening disorder, because edema occurring in the mucosa of the upper airways can lead to suffocation. The management of <jats:styled-content style="fixed-case">HAE</jats:styled-content> consists of avoiding the triggering factors, prophylaxis, and the acute treatment of edematous episodes. Medical procedures can also provoke edematous attacks, and therefore, short‐term prophylaxis (<jats:styled-content style="fixed-case">STP</jats:styled-content>) is recommended before such interventions. Our aim was to evaluate the efficacy and safety of <jats:styled-content style="fixed-case">STP</jats:styled-content> administered before medical procedures.</jats:p></jats:sec><jats:sec><jats:title>Methods</jats:title><jats:p>We conducted a retrospective analysis before and a prospective survey after establishing the diagnosis in a group of 137 (60 males, 77 females; 20 pediatric and 117 adult) patients with <jats:styled-content style="fixed-case">HAE</jats:styled-content>. Both were implemented using questionnaires, patient diaries and hospital charts focusing on medical interventions provoking edematous attack, and the medicinal products (<jats:styled-content style="fixed-case">C</jats:styled-content>1‐<jats:styled-content style="fixed-case">INH</jats:styled-content> concentrate, tranexamic acid, and danazol) administered for <jats:styled-content style="fixed-case">STP</jats:styled-content>.</jats:p></jats:sec><jats:sec><jats:title>Results</jats:title><jats:p>Comparing surgical interventions performed without pre‐event <jats:styled-content style="fixed-case">STP</jats:styled-content> (in 39/89 patients before <jats:styled-content style="fixed-case">HAE</jats:styled-content> was diagnosed), or after <jats:styled-content style="fixed-case">STP</jats:styled-content> (in 3/55 cases after diagnosis), we found a significant (<jats:italic>P</jats:italic> < 0.0001, Fisher's exact test) reduction in the number of edematous episodes. Evaluating the efficacy of the drugs administered for <jats:styled-content style="fixed-case">STP</jats:styled-content> revealed that <jats:styled-content style="fixed-case">C</jats:styled-content>1‐<jats:styled-content style="fixed-case">INH</jats:styled-content> concentrate (Berinert<jats:sup>®</jats:sup>, CSL Behring, Marburg, Germany) was significantly (<jats:italic>P</jats:italic> = 0.0096, Fisher's exact test) superior to orally administered drugs in reducing the instances of postprocedural edema. None of the medicinal products caused adverse events potentially related to <jats:styled-content style="fixed-case">STP</jats:styled-content>.</jats:p></jats:sec><jats:sec><jats:title>Conclusions</jats:title><jats:p><jats:styled-content style="fixed-case">STP</jats:styled-content> reduces the number of postprocedural edematous episodes. <jats:styled-content style="fixed-case">C</jats:styled-content>1‐<jats:styled-content style="fixed-case">INH</jats:styled-content> concentrate is safe and effective for prophylaxis. When this agent is not available, danazol is a potential alternative for prophylaxis before elective medical interventions.</jats:p></jats:sec>