Berotralstat Improves Quality of Life in Hereditary Angioedema

Patients with hereditary angioedema taking berotralstat prophylactically had improved quality of life, according to the results of a recent study presented at the American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, held virtually from November 13 to 15.

Researchers evaluated quality of life via the Angioedema Quality of Life (AE-QoL) questionnaire in adult patients with hereditary angioedema as part of an analysis of the phase 3 APeX-2 study (ClinicalTrials.gov Identifier: NCT03485911) Results were compared between groups of patients taking berotralstat, a plasma kallikrein inhibitor (110 mg/d or 150 mg/d), and a control group taking placebo after 24 weeks and 48 weeks. At week 24, patients who were initially assigned to berotralstat 110 mg per day or 150 mg per day continued on the same dose for an additional 24 weeks. The minimally clinically important difference was determined to be 6 points on the questionnaire. Decreases in score represented an increase in QoL.

A total of 121 patients with hereditary angioedema were included in the study. The 150 mg per day berotralstat group saw a mean decrease from baseline of -15 points. The 6-point minimally clinically important difference threshold was exceeded after the first month and sustained through week 48. All 4 domains — functioning, fatigue/mood, fear/shame, and nutrition — demonstrated improvement. The functional domain represented the largest improvement (mean change from baseline, -24).

“Reductions in [hereditary angioedema] attacks during berotralstat therapy are associated with clinically important improvements in QoL,” the study authors concluded.

Disclosure: This clinical trial was supported by BioCryst Pharmaceuticals. Please see the original reference for a full list of authors’ disclosures.

Reference

Johnston D, Banerji A, Riedl M, et al. Berotralstat improves patient-reported quality of life through 48 weeks in the phase 3 Apex-2 Trial. Presented at: the American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting (Virtual Experience); November 13-15, 2020. Abstract P154.