Successful long-term control of lanadelumab-refractory type 2 hereditary angioedema with berotralstat

Abstract

Type 2 hereditary angioedema (HAE-C1INH-Type2) is a rare, potentially life-threatening disorder characterized by functional impairment of C1-esterase inhibitor (C1-INH). Responses to long-term prophylactic (LTP) therapies are variable. We describe a case of HAE-C1INH-Type2 refractory to tranexamic acid, danazol, C1-INH concentrate, and lanadelumab, successfully controlled with berotralstat. A 47-year-old woman with HAE-C1INH-Type2 experienced recurrent facial, extremity, and laryngeal angioedema attacks. Danazol 400 mg/day provided initial control but caused severe dyslipidemia and transaminase elevation. Sequential interventions (tranexamic acid, lanadelumab, C1-INH concentrate) resulted in suboptimal control, with recurrent laryngeal attacks requiring frequent icatibant. Berotralstat 150 mg/day achieved complete and sustained remission with improvement in Angioedema control Test (AECT), Angioedema Quality of Life (AE-QoL) and Treatment Satisfaction Questionnaire for Medication II (TSQM-II) scores. Despite sharing therapeutic targets, berotralstat achieved superior disease control and quality of life in this patient compared to previous LTP treatments, representing a highly effective alternative following lanadelumab failure.