Short-term prophylaxis for children and adolescents with hereditary angioedema

Abstract

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.

The international WAO/EAACI guideline for the management of hereditary angioedema—The 2021 revision and update

“…For scheduled preprocedural prophylaxis, androgens are used for 5 days before and 2–3 days postevent. Tranexamic acid has been used for preprocedural prophylaxis in the past; however, it is not recommended by most guideline experts 7,11,169,173–175 …”

The international WAO/EAACI guideline for the management of hereditary angioedema – The 2021 revision and update

“…Fresh frozen plasma (FFP) may be used for short-term prophylaxis, but it is not as safe as intravenous pdC1-INH concentrate and is a second-line agent because of the greater risk of blood borne disease transmission and allosensitization. 7 , 11 , 169 , 173 , 174 , 175 , 176 …”

“…For scheduled pre-procedural prophylaxis, androgens are used for 5 days before and 2–3 days post event. Tranexamic acid has been used for pre-procedural prophylaxis in the past, however it is not recommended by most guideline experts 7 , 11 , 169 , 173 , 174 , 175 …”

“…With all pre-procedural prophylactic treatments, break-through attacks can occur, so patients and treating physicians should be aware of this increased risk and understand the treatment plan, and on demand treatment needs to be available. 11 , 165 , 166 , 173 , 176 …”

Hereditary Angioedema

“…Several observational studies have shown that use of short-term prophylaxis during high-risk procedures is effective in preventing an attack of angioedema. [ 34 35 36 37 ]…”