Canadian hereditary angioedema guideline

Abstract

Hereditary angioedema (HAE) is a disease which is associated with random and often unpredictable attacks of painful swelling typically affecting the extremities, bowel mucosa, genitals, face and upper airway. Attacks are associated with significant functional impairment, decreased Health Related Quality of Life, and mortality in the case of laryngeal attacks. Caring for patients with HAE can be challenging due to the complexity of this disease. The care of patients with HAE in Canada is neither optimal nor uniform across the country. It lags behind other countries where there are more organized models for HAE management, and where additional therapeutic options are licensed and available for use. The objective of this guideline is to provide graded recommendations for the management of patients in Canada with HAE. This includes the treatment of attacks, short-term prophylaxis, long-term prophylaxis, and recommendations for self-administration, individualized therapy, quality of life, and comprehensive care. It is anticipated that by providing this guideline to caregivers, policy makers, patients and their advocates, that there will be an improved understanding of the current recommendations regarding management of HAE and the factors that need to be considered when choosing therapies and treatment plans for individual patients. The primary target users of this guideline are healthcare providers who are managing patients with HAE. Other healthcare providers who may use this guideline are emergency physicians, gastroenterologists, dentists and otolaryngologists, who will encounter patients with HAE and need to be aware of this condition. Hospital administrators, insurers and policy makers may also find this guideline helpful.Electronic supplementary materialThe online version of this article (doi:10.1186/1710-1492-10-50) contains supplementary material, which is available to authorized users.

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.…”

“…As in adults, indications for STP in pediatrics include patient‐specific triggers, medical and dental procedures 85. For most ‘minor interventions’, the recommendation is to choose on‐demand treatment if a swelling event is precipitated rather than prophylaxis, provided that a licensed on‐demand medication is immediately available in the case of emergency (level III evidence).…”

“…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.…”