Hereditary angioedema (HAE) in children and adolescents—a consensus on therapeutic strategies

Abstract

Hereditary angioedema due to C1 inhibitor (C1 esterase inhibitor) deficiency (types I and II HAE-C1-INH) is a rare disease that usually presents during childhood or adolescence with intermittent episodes of potentially life-threatening angioedema. Diagnosis as early as possible is important to avoid ineffective therapies and to properly treat swelling attacks. At a consensus meeting in June 2011, pediatricians and dermatologists from Germany, Austria, and Switzerland reviewed the currently available literature, including published international consensus recommendations for HAE therapy across all age groups. Published recommendations cannot be unconditionally adopted for pediatric patients in German-speaking countries given the current approval status of HAE drugs. This article provides an overview and discusses drugs available for HAE therapy, their approval status, and study results obtained in adult and pediatric patients. Recommendations for developing appropriate treatment strategies in the management of HAE in pediatric patients in German-speaking countries are provided.Conclusion Currently, plasma-derived C1 inhibitor concentrate is considered the best available option for the treatment of acute HAE-C1-INH attacks in pediatric patients in German-speaking countries, as well as for short-term and long-term prophylaxis.

International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency

“…So far, no randomized controlled trials (RCTs) on prophylactic treatment restricted to the pediatric population have been conducted. Few pediatric patients have been included in RCTs (no level I evidence) leaving most pediatric prophylaxis level III evidence 14, 21, 22, 34, 38, 78, 83, 84, 85.…”

“…For interventions that involve airway manipulation or that might lead to tissue swelling, prophylaxis with a dose of 15 to 30 units per kg pdC1‐INH (Berinert ® [pdC1‐INH Be ]) concentrate is recommended. There are no studies supporting appropriate timing of the STP nor consensus on the recommended maximum dose (1000 units versus 15 to 30 units/kg) (level III evidence) 14, 21, 22, 34, 78, 83, 84, 85. STP with pdC1‐INH recommendations varies from during procedure or one or more hours before the procedure trying to give as close to the procedure as possible.…”

“…Prophylaxis should start (at least) 5 days before and be continued for 2 days postprocedure (level III evidence). As prophylaxis may fail, effective on‐demand treatment should be available whenever possible (level III evidence) 14, 21, 22, 34, 78, 83, 84, 85. In emergency situations and when licensed on‐demand therapies are not available, 10 ml/kg of solvent detergent plasma (SDP) (safer than fresh frozen plasma (FFP)) may be used prophylactically pre‐ or perioperatively or on‐demand (level III evidence).…”

“…Combination LTP approaches including intermittent LTP or combination LTP agents (e.g., TA plus pdC1‐INH at various doses and frequencies) need further consideration. To date, safety, efficacy, and tolerability of pdC1‐INH appear to be similar in pediatric and adult patients (level III evidence), but approval age and indication of various pdC1‐INH concentrates vary by jurisdiction 14, 21, 22, 34, 78, 83, 84, 85, 88, 89.…”

Hereditary Angioedema in Children — A Rare Clinical Case in the Practice of the Abdominal Surgeon

“…Достоверные данные о распространенности заболевания среди населения Российской Федерации отсутствуют. Наследственный ангионевротический отек представляет серьезную проблему в практической медицине в связи с тяжестью обострений и высокой вероятностью разви-тия тяжелых осложнений, которые в некоторых случаях приводят к летальному исходу [7,8].…”