Berotralstat Tolerability and Effectiveness in Hereditary Angioedema: Berolife Study Results
Abstract
BACKGROUND: Berotralstat is a first-line once-daily oral prophylactic treatment for hereditary angioedema (HAE). Berolife was designed to evaluate the tolerability and effectiveness of berotralstat in real-world conditions. METHODS: Berolife is an open-label, multicenter, observational study conducted in France from September 2021 to January 2024. The primary objective was to assess tolerability, and secondary objectives included characterization of the treated population and assessment of berotralstat effectiveness. HAE attack rate was assessed before and after initiation of berotralstat using the paired Wilcoxon rank-sum test. RESULTS: Altogether, 80 patients were enrolled in the study (75 with HAE-C1INH type 1 or 2 and 5 with HAE-nC1INH) and received at least one dose of berotralstat. At baseline, patients had a mean (standard deviation [SD]) age of 40.0 (17.5) years, and most (67.5%) had received prior long-term prophylaxis treatment. The mean (SD) duration of berotralstat treatment was 11.5 (8.5) months. Treatment-related adverse events (AEs) occurred in 45.0% of patients, with gastrointestinal disorders being the most common (diarrhea: 13.8%, abdominal pain: 12.5%, upper abdominal pain: 7.5%). Importantly, no treatment-related serious AEs were reported. A total of 61 patients were treated with berotralstat for ≥ 6 months and evaluated for effectiveness. At 6 months of treatment, a significant reduction in monthly HAE attack rates was observed (mean [SD]: 0.62 [0.66] vs. mean [SD]: 1.25 [1.10] at baseline; p = 0.001). CONCLUSIONS: Berotralstat was generally well tolerated with a tolerability profile consistent with prior clinical trials. Significant reductions in monthly HAE attack rates were observed, confirming its effectiveness in real-world settings.
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BibTeX
@article{gobert2026berotralstat,
title = {{Berotralstat Tolerability and Effectiveness in Hereditary Angioedema: Berolife Study Results}},
author = {Delphine Gobert and David Launay and Isabelle Boccon-Gibod and Melisande Bourgoin-Heck and Cyrille Hoarau and Yann Ollivier and Hervé Maillard and Fabien Pontille and Nicolas Ozanne and Magali Aubineau and Claire de Moreuil and Anne Gerber and Fabien Pelletier and Pierre‐yves Jeandel and Aurélie Du-Thanh and Marie‐caroline Dalmas and Marie‐caroline Taquet and Jean Schmidt and Amélie Servettaz and Amandine Perier and Enzo Cohen and Laure Carriat and Nicolas Lemaire and Mona Villedieu and Jean‐charles Crave and Olivier Fain and Laurence Bouillet},
journal = {Clinical and Translational Allergy},
year = {2026},
month = aug,
doi = {10.1002/clt2.70193},
url = {https://hal.science/hal-05735422v1},
note = {CC BY},
}
RIS
TY - JOUR
TI - Berotralstat Tolerability and Effectiveness in Hereditary Angioedema: Berolife Study Results
AU - Delphine Gobert
AU - David Launay
AU - Isabelle Boccon-Gibod
AU - Melisande Bourgoin-Heck
AU - Cyrille Hoarau
AU - Yann Ollivier
AU - Hervé Maillard
AU - Fabien Pontille
AU - Nicolas Ozanne
AU - Magali Aubineau
AU - Claire de Moreuil
AU - Anne Gerber
AU - Fabien Pelletier
AU - Pierre‐yves Jeandel
AU - Aurélie Du-Thanh
AU - Marie‐caroline Dalmas
AU - Marie‐caroline Taquet
AU - Jean Schmidt
AU - Amélie Servettaz
AU - Amandine Perier
AU - Enzo Cohen
AU - Laure Carriat
AU - Nicolas Lemaire
AU - Mona Villedieu
AU - Jean‐charles Crave
AU - Olivier Fain
AU - Laurence Bouillet
PY - 2026
DA - 2026/08/15
JO - Clinical and Translational Allergy
DO - 10.1002/clt2.70193
UR - https://hal.science/hal-05735422v1
AB - BACKGROUND: Berotralstat is a first-line once-daily oral prophylactic treatment for hereditary angioedema (HAE). Berolife was designed to evaluate the tolerability and effectiveness of berotralstat in real-world conditions. METHODS: Berolife is an open-label, multicenter, observational study conducted in France from September 2021 to January 2024. The primary objective was to assess tolerability, and secondary objectives included characterization of the treated population and assessment of berotralstat effectiveness. HAE attack rate was assessed before and after initiation of berotralstat using the paired Wilcoxon rank-sum test. RESULTS: Altogether, 80 patients were enrolled in the study (75 with HAE-C1INH type 1 or 2 and 5 with HAE-nC1INH) and received at least one dose of berotralstat. At baseline, patients had a mean (standard deviation [SD]) age of 40.0 (17.5) years, and most (67.5%) had received prior long-term prophylaxis treatment. The mean (SD) duration of berotralstat treatment was 11.5 (8.5) months. Treatment-related adverse events (AEs) occurred in 45.0% of patients, with gastrointestinal disorders being the most common (diarrhea: 13.8%, abdominal pain: 12.5%, upper abdominal pain: 7.5%). Importantly, no treatment-related serious AEs were reported. A total of 61 patients were treated with berotralstat for ≥ 6 months and evaluated for effectiveness. At 6 months of treatment, a significant reduction in monthly HAE attack rates was observed (mean [SD]: 0.62 [0.66] vs. mean [SD]: 1.25 [1.10] at baseline; p = 0.001). CONCLUSIONS: Berotralstat was generally well tolerated with a tolerability profile consistent with prior clinical trials. Significant reductions in monthly HAE attack rates were observed, confirming its effectiveness in real-world settings.
SN - 2045-7022
ER -