Efficacy of oral long-term prophylaxis over 6 months with Berotralstat as off-label use for patients with acquired angioedema (AAE) – real world data from two study centers

Introduction  Hereditary angioedema (HAE) due to genetic C1-Esterase-Inhibitor (C1-INH) deficiency is a rare disease with an incidence of about 1,5:100.000. There are several licensed treatments available. The acquired C1-INH deficiency in AAE is 9-fold rarer. If no underlying – often malignant – condition is found to be treated, there is no licensed therapy available.

Methods  The data of all AAE patients referring to two angioedema centers in southern Germany was analyzed. Inclusion criteria were lacking underlying disease or no need of treatment for the condition, insufficient control of disease with acute medication and off-label long-term oral prophylaxis with Berotralstat. Changes in quality of life (AE-QoL angioedema quality of life) and control of disease (AECT angioedema control test) were measured with validated questionnaires prior and after therapy with Berotralstat.

Results 3 patients with AAE have been included. A mean improvement of 9 points in AE-QoL after only 2 months and of 23 points after 6 months could be found, with a change of 6 points given as significant. The AECT improved by a mean of 3,5 points after 2 months and 6,5 points after a total of 6 months, reaching a mean of 13 points. The highest reachable score is 16, the cut-off for good disease control is ≥10 points and was reached by all three patients.

Conclusion In AAE-patients with no other therapy options and poor disease control under solely acute therapy, off-label oral long-term prophylaxis with Berotralstat resulted in relevant improvement of quality of life and symptom relief.

Publication History

Article published online:
12 May 2023