Clinical experience with berotralstat in patients with hereditary angioedema: an Italian case series from the ITACA cohort

SUMMARY

Background. Recent advancements in hereditary angioedema (HAE) management have focused mainly on long-term prophylaxis (LTP), aiming to achieve total disease control and normalize patients’ lives. The latest international guidelines recommend as first-line options for LTP plasma-derived C1-INH, lanadelumab, and berotralstat. Berotralstat is a highly selective inhibitor of plasma kallikrein and the first licensed oral therapy for HAE, to be taken once daily. Given the limited clinical experience with berotralstat in Italy, members of the Italian Network for Hereditary and Acquired Angioedema (ITACA) here present and discuss some representative clinical cases treated with berotralstat in their institutions to provide practical guidance for the use of this medication. Methods. Three main topics were identified, (I) the switch from injectable medications to new oral therapies, with a focus on shared decision-making, (II) the switch from androgens to berotralstat, and (III) the management of comorbid patients, with a focus on polypharmacy. Results. The Italian experience to date indicates that (I) the decision on LTP should be always shared with patients and, given the importance for patients of administration route, berotralstat should always be considered and proposed; (II) an overlap period between androgens and berotralstat, with progressive tapering of androgens,  is the more appropriate strategy when planning this shift; (III) potential drug-drug interactions must be evaluated in patients on multiple medications, with berotralstat showing a good safety profile in this perspective. Conclusions. Patient engagement, awareness of androgen risks, transition strategies and drug-interaction evaluation are essential to guide individualized LTP choices.

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