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Review
. 2023 Dec;65(3):354-364.
doi: 10.1007/s12016-023-08972-2. Epub 2023 Nov 2.

A Retrospective Analysis of Long-Term Prophylaxis with Berotralstat in Patients with Hereditary Angioedema and Acquired C1-Inhibitor Deficiency-Real-World Data

Felix Johnson  1 , Anna Stenzl  2 , Benedikt Hofauer  1 , Helen Heppt  1 , Eva-Vanessa Ebert  3 , Barbara Wollenberg  3 , Robin Lochbaum  4 , Janina Hahn  4 , Jens Greve  4 , Susanne Trainotti  3
Affiliations
Review

A Retrospective Analysis of Long-Term Prophylaxis with Berotralstat in Patients with Hereditary Angioedema and Acquired C1-Inhibitor Deficiency-Real-World Data

Felix Johnson et al. Clin Rev Allergy Immunol. 2023 Dec.

Abstract

Hereditary angioedema (HAE) and acquired C1-inhibitor deficiency (AAE-C1-INH) are orphan diseases. Berotralstat is a recently licensed long-term prophylaxis (LTP) and the first oral therapy for HAE patients. No approved therapies exist for AAE-C1-INH patients. This study is the first to report real-world clinical data of patients with AAE-C1-INH and HAE who received Berotralstat. All patients treated with Berotralstat were included in this retrospective, bi-centric study. Data was collected from patients' attack calendars and the angioedema quality of life (AE-QoL) and angioedema control test (AECT) questionnaires before treatment, and at 3, 6, and 12 months after treatment and was then analyzed. Twelve patients were included, 3 patients with AAE-C1-INH, 7 patients with HAE type I, and 2 patients with HAE-nC1-INH. One patient (HAE I) quit treatment. Berotralstat was associated with fewer attacks in all groups. After 6 months of treatment, a median decrease of attacks per month was noted for HAE type I patients (3.3 to 1.5) and AAE-C1-INH patients (2.3 to 1.0). No aerodigestive attacks were noted for AAE-C1-INH patients. For HAE-nC1-INH patients, a mean decrease from 3.8 to 1.0 was noted (3 months). For HAE I patients, the total AE-QoL lowered a mean of 24.1 points after 6 months, for HAE-nC1-HAE patients 8.0 points, and for AAE-C1-INH patients 13.7 points. AECT scores increased for HAE I patients (mean: 7.1), HAE-nC1-INH patients (9.0), and AAE-C1-INH patients (4.2) after 6 months. Patients with HAE, HAE-nC1-INH, and AAE-C1-INH treated with Berotralstat showed reduced angioedema attacks and improved AE-QoL and AECT scores.

Keywords: AAE-C1-INH; Acquired angioedema; Berotralstat; Bradykinin; C1-INH-deficiency; HAE; Hereditary angioedema; LTP; Long-term prophylaxis; Prophylaxis.

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Conflict of interest statement

No financial assistance from any pharmaceutical company was obtained for this study. The authors of this study declare the following conflict of interests: F. Johnson: has received grant research support and/or speaker/consultancy fees from CSL Behring, Takeda and Pharming. He has also received funding to attend conferences/educational events from CSL Behring, Takeda and Pharming. He has participated as an investigator in a clinical trial/registry for CSL Behring, BioCryst, IONIS Pharmaceuticals and Takeda. A. Stenzl: has no conflict of interest to declare regarding this publication B. Hofauer: has participated as an investigator in clinical trials for CSL Behring and Takeda. H. Heppt: has no conflict of interest to declare regarding this publication E. Ebert: has received grant research support and/or speaker/consultancy fees from CSL Behring and Takeda. She has also received funding to attend conferences/educational events from CSL Behring, Takeda and Pharming. She has participated as an investigator in a clinical trial/registry for CSL Behring, BioCryst, IONIS Pharmaceuticals and Takeda. J. Hahn: has received speaker/consultancy fees from CSL Behring and Takeda. She has also received funding to attend conferences/educational events from CSL Behring and Takeda. She has participated as an investigator in a clinical trial/registry for CSL Behring, BioCryst, Pharvaris and Takeda. J. Greve: has received speaker/consultancy fees from CSL Behring, Takeda, Kalvista and BioCryst. He has also received funding to attend conferences/educational events from CSL Behring and Takeda. He has participated as an investigator in a clinical trial/registry for CSL Behring, BioCryst and Takeda. B. Wollenberg: has no conflict of interest to declare regarding this publication R. Lochbaum: Robin Lochbaum has received consultancy fees from BioCryst and Takeda. He has also received funding to attend conferences/educational events from CSL Behring, BioCryst and Takeda. He has participated as an investigator in a clinical trial/registry for Takeda and Pharvaris. S. Trainotti: has received grant research support and/or speaker/consultancy fees from CSL Behring and Takeda. She has also received funding to attend conferences/educational events from CSL Behring and Takeda. She has participated as an investigator in a clinical trial/registry for CSL Behring, BioCryst, IONIS Pharmaceuticals and Takeda.

Figures

Fig. 1
Fig. 1
Total attacks before and after LTP with Berotralstat for AAE-C1-INH patients (n = 3)
Fig. 2
Fig. 2
Monthly attack rate before and after LTP with Berotralstat for AAE-C1-INH patients (n = 3)
Fig. 3
Fig. 3
Attack rate per domain before and after LTP with Berotralstat for AAE-C1-INH patients (n = 3)
Fig. 4
Fig. 4
Total AE-QoL for all the patients AAE-C1-INH and HAE (n = 12) over the whole observation period
Fig. 5
Fig. 5
Analysis of AE-QoL scores pre-dose and after 6 months using Wilcoxon matched pairs test signed rank test (p < 0.0449)
Fig. 6
Fig. 6
Total AECT for all the patients AAE-C1-INH and HAE (n = 12) over the whole observation period
Fig. 7
Fig. 7
Analysis of AECT scores pre-dose and after 6 months using Wilcoxon matched pairs test signed rank test (p < 0.0289)
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