Short-term prophylaxis for children and adolescents with hereditary angioedema

Background: Hereditary Angioedema (HAE) is a rare, autosomal dominant, life threatening disease, secondary to the deficiency of C1-inhibitor, dysfunction of C1-inhibitor or inadequate control of the contact pathway. Presentation includes recurrent swelling of the skin, upper airway and the abdomen. Trauma can precipitate attacks, which in the airway can lead to asphyxia. For this reason, short term prophylaxis (STP) may be indicated before medical, surgical and dental procedures. The goal of the manuscript is to review short term prophylaxis for children of all ages. Methods: We searched the following search words: children, pediatric, adolescent, plasma derived C1-inhibitor, recombinant C1-inhibitor, surgery, medical procedures, prophylaxis, dental, Hereditary Angioedema, tranexamic acid, androgens, fresh frozen plasma, short term prophylaxis, lanadelumab, subcutaneous C1-inhibitor in Google Scholar and in PubMed to develop our results. Results: STP should be discussed at every visit. Plans should be individualized based upon the procedure, therapies available and shared decision making with patient/parent. For high risk procedures plasma derived C1-inhibitor should be used at 20 units/ kg just prior to the procedure. Alternative agents for STP include recombinant C1-inhibitor, fresh frozen plasma, androgens, or tranexamic acid. In all cases, with or without the use of STP, 2 doses of on-demand therapy should be available in case of an attack. Conclusion: Herein, we review the published data on STP for pediatric patients with HAE and discuss first-line options, and off label use of medications, as well as review the guidelines pertaining to short term prophylaxis.