Establishing the ultimate goals of treatment in hereditary angioedema

Published: May 24, 2021

Hereditary angioedema (HAE) is a rare, life-threatening disorder associated with recurrent episodes of swelling. Swelling can occur in various parts of the body including the hands, feet, face and airway. These attacks are painful and unpredictable and have a profound impact on patient quality of life. In most patients, HAE is caused by a deficiency in C1-inhibitor protein, and disease management for patients with HAE is currently achieved through a combination of on-demand ‘rescue’ medication and short- and long-term prophylaxis. Recent advances in long-term prophylaxis, which include subcutaneously administered plasma-derived C1-inhibitor protein and the monoclonal antibody lanadelumab, provide both increased efficacy and decreased treatment burden.

In a recent article in The Journal of Allergy and Clinical Immunology (JACI), Maurer et al. aimed to establish the goals of treatment for this disabling disease, and to define the factors that should be considered when assessing disease control and quality of life for patients with HAE. To achieve this, a Delphi process was used to obtain the opinions of a global panel of experts. The Delphi method is an approach that uses several rounds of questioning to arrive or converge at a group opinion or consensus. The anonymity of this process is key and ensures that all opinions are considered equally. In this case, three rounds of voting were used, and consensus was considered to be reached if at least 75% of respondents agreed with the statement. The authors hope that the results of this initiative will provide benchmarks for assessing disease control and treatment efficacy, and ultimately lead to improvements in disease management for patients with HAE.

The Delphi process revealed that the ultimate goals of HAE treatment are to achieve total control of the disease (ie, no attacks) and to normalize the patient’s life. In addition to these goals, the panel also agreed that a further 18 factors should be considered when assessing disease control and patient quality of life. The agreed factors were wide-ranging and included, for example, the need for rescue medication, the number of attacks in a given time period, the number of hours of activity impairment and the number of emergency department or hospital visits. The authors noted that the number of agreed consensus statements may reflect variability in access to highly effective therapies and specialized disease management across the globe.

Overall, this consensus supports taking a holistic, patient-centered and shared decision-making approach when assessing disease control and patient quality of life. It also established ‘treat-to-target’ style goals for HAE, the ultimate goals being to achieve no attacks and to normalize the patient’s life. The availability of improved treatment and disease management means that complete control of HAE is now becoming a realistic possibility for some patients.

The Journal of Allergy and Clinical Immunology (JACI) is an official scientific journal of the AAAAI, and is the most-cited journal in the field of allergy and clinical immunology.

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