Treatment of Laryngeal Hereditary Angioedema

Characterization of prodromal symptoms in a large population of patients with hereditary angio-oedema

“…The angio‐oedema may occur in variable degrees of severity, and it is often localized to one or several locations of the body including the limbs, genitalia, face, oropharynx, tongue, laryngeal tissues, torso and visceral organs . Because of its significant morbidity and mortality of 15–30% due to untreated or insufficiently treated laryngeal oedema and subsequent asphyxiation, all patients need to carry emergency therapies, and some require long‐term prophylaxis …”

Plasma‐derived C1‐INH for managing hereditary angioedema in pediatric patients: A systematic review

“…The use of pdC1‐INH for long‐term prophylaxis (LTP) in pediatric patients was reported in one small placebo‐controlled study , an open‐label study , a small prospective cohort study , two retrospective cohort studies , and two case reports , all of which reported low attack rates in children using pdC1‐INH. The one randomized, placebo‐controlled study was a 24‐week crossover study involving four children between the ages of 9 and 17 years .…”

“…On‐demand therapy refers to the administration of an HAE agent to terminate the symptoms of acute attacks as they occur. The use of pdC1‐INH as on‐demand therapy in pediatric patients with HAE was reported in two placebo‐controlled trials (described in three publications) , four prospective open‐label studies , five retrospective cohort studies , and one case report (Table S2). The dosing of pdC1‐INH in the various reports varied by specific pdC1‐INH product (Cinryze, fixed dose vs. Berinert, weight‐based dose) and also by date of the study for Berinert.…”

“…Those identified from abstracts as potentially containing data describing pdC1-INH use in children were retrieved in full-text format and further screened. This process identified 15 publications in which pdC1-INH was used for the treatment of HAE in pediatric patients (18,(23)(24)(25)(26)(27)(28)(29)(30)(31)(32)(33)(34)(35)(36). In the process of reviewing these 15 citations and their bibliographies, two additional case reports were identified (37,38).…”

“…On-demand therapy refers to the administration of an HAE agent to terminate the symptoms of acute attacks as they occur. The use of pdC1-INH as on-demand therapy in pediatric patients with HAE was reported in two placebo-controlled trials (described in three publications) (23,24,36), four prospective open-label studies (23,25,27,36), five retrospective cohort studies (29,(31)(32)(33)39), and one case report (35) ( Table S2) achieved unequivocal relief within 4 h after dosing compared with those treated with placebo (71% vs. 40%) (23). One pediatric patient treated with pdC1-INH did not achieve unequivocal relief within 4 h, but symptoms were fully resolved at 11.75 h after treatment.…”