Berotralstat in hereditary angioedema due to C1 inhibitor deficiency: first real-world evidence from a Canadian center

Abstract

BackgroundHereditary angioedema due to C1 inhibitor deficiency is a rare genetic condition that causes recurrent swelling with consequent functional impairment and decreased quality of life. Long-term prophylaxis (LTP) to prevent angioedema episodes is a key component of disease management. Berotralstat, an oral, once-daily plasma kallikrein inhibitor, was approved for LTP by Health Canada in 2022.MethodsWe conducted a retrospective, real-world study investigating the effectiveness and adverse effects of berotralstat. Data on angioedema frequency, disease control, and adverse events were tabulated. Patient satisfaction with treatment was scored on a 5-point Likert scale, with 1 representing very unsatisfied and 5 representing very satisfied with therapy.ResultsFrom June, 2022 and May, 2023, 8 patients with HAE type 1 or type 2 received berotralstat. Effectiveness data were available for 7 patients who continued the drug for at least 3 months, 4 of whom switched to berotralstat from plasma-derived C1 inhibitor LTP. In these 7 patients, the average number of attacks per month decreased from 3.3 to 1.6 (p<0.05), representing a ~52% reduction in attack frequency. Median angioedema control test score numerically improved from 8 to 13 (p=0.0781). Of the 8 patients who received berotralstat, 3 reported no adverse effects and 5 experienced gastrointestinal side effects, which were mild and transient in 3 and led to discontinuation in 1. Average treatment satisfaction was between satisfied and very satisfied at 4.3.ConclusionBerotralstat is an effective agent for long-term prophylaxis in HAE. Most patients experienced no adverse effects or mild, transient gastrointestinal symptoms.

Centralized care model for hereditary angioedema overcomes geographical barriers

“…Canadian Blood Services provides access to subcutaneous pd-C1 LTP (Haegarda/Berinert 2000) to all patients irrespective of private insurance coverage. Lanadelumab and berotralstat were also available for most patients and there is significant local experience in using these drugs as first line LTP therapies ( 19 , 20 ). Those residing in extremely remote regions can be provided with transportation services funded by territorial health systems to attend appointments.…”