In patients with hereditary angioedema (HAE), long-term prophylaxis with berotralstat was associated with sustained, clinically meaningful improvements in patient-reported quality of life (QoL), according to an analysis of patients treated with the agent in the APeX-2 trial (NCT03485911).

Results of the study will be presented at the 2023 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting, which is scheduled to be held in San Antonio, Texas, February 24-27, 2023, and have been published in The Journal of Allergy and Clinical Immunology.

Berotralstat is a first-line, once-daily oral prophylactic treatment for patients with HAE that has demonstrated effectiveness in reducing the burden of disease and treatment. In the current study, QoL was evaluated using the validated Angioedema-QoL questionnaire (AE-QoL), which was stratified according to sex, baseline age, baseline rate of attacks, prior prophylaxis, and incidence of gastrointestinal adverse events (GI AEs) while receiving berotralstat.

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Decreasing AE-QoL scores were indicative of improvements in QoL. The minimal clinically important difference (MCID) was defined as a 6-point reduction in total AE-QoL score.

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Results of the study showed that among the 40 participants treated with 150 mg of berotralstat once daily, the mean improvement in total AE-QoL score from baseline to week 96 exceeded the MCID value from week 4 and was sustained through week 96 when stratified by age (<35 years, –23.6; 35-50 years, –21.4; >50 years, –24.5); sex (female, –23.9; male, –21.7); baseline attack rate (<2 per month, –24.7; ≥2 per month, –22.4); and prior prophylaxis (prior androgen therapy, –19.4; prior C1 inhibitor therapy, –21.6).

The mean total AE-QoL score improved from baseline to week 96 regardless of the presence (n=11; –18.2) or absence (n=8; –29.5) of GI AEs during the initial 24 weeks of berotralstat therapy. Improvements were observed across all QoL domains (ie, functioning, fatigue/mood, fear/shame, and nutrition) over 96 weeks, regardless of stratification.

The authors concluded that “long-term prophylaxis with berotralstat led to sustained and clinically meaningful improvements in patient-reported QoL across multiple subgroups, suggesting sustained reductions in disease and treatment burden.”


Soteres D, Lumry W, Magerl M, et al. Berotralstat improved quality of life through 96 weeks across multiple subgroups of patients with hereditary angioedema. J Allergy Clin Immunol. 2023;151(2):AB139. doi:10.1016/j.jaci.2022.12.434