Hereditary angioedema outcomes in US patients switched from injectable long-term prophylactic medication to oral berotralstat.
APeX-S
Berotralstat
Hereditary angioedema
Injectable prophylaxis
Oral prophylaxis
Prophylactic treatments
Safety
Treatment satisfaction
Treatment switch
Journal
Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology
ISSN: 1534-4436
Titre abrégé: Ann Allergy Asthma Immunol
Pays: United States
ID NLM: 9503580
Informations de publication
Date de publication:
23 Nov 2023
23 Nov 2023
Historique:
received:
11
08
2023
revised:
30
10
2023
accepted:
17
11
2023
medline:
26
11
2023
pubmed:
26
11
2023
entrez:
25
11
2023
Statut:
aheadofprint
Résumé
Berotralstat, a first-line, once-daily oral plasma kallikrein inhibitor for long-term prophylaxis of hereditary angioedema (HAE), is an effective and well-tolerated treatment option. This report summarizes the safety, effectiveness, and impact on treatment satisfaction in patients who switched from injectable long-term prophylactics (LTPs) to oral berotralstat monotherapy (150 mg daily) at US sites in the international open-label APeX-S study. APeX-S (ClinicalTrials.gov, NCT03472040) was an open-label Phase 2 study of berotralstat conducted in 22 countries. Here, we focus on APeX-S patients enrolled at US sites who switched from injectable LTPs to berotralstat 150 mg once-daily monotherapy. Thirty-four patients discontinued lanadelumab (n = 21), subcutaneous C1 esterase inhibitor (n = 11), or intravenous C1 esterase inhibitor (n = 2) and switched to berotralstat 150 mg monotherapy. Vomiting, diarrhea, and upper respiratory tract infection were the most common adverse events (each 11.8%). Mean monthly attack rates were consistently low following the switch to berotralstat. The mean (standard error of the mean) monthly attack rate was 0.29 (0.11) at Month 1, 0.48 (0.15) at Month 6, and 0.58 (0.23) at Month 12. The median attack rate was 0 attacks/month throughout 12 months of treatment. Improvements were observed in the Treatment Satisfaction Questionnaire for Medication from baseline to Month 12 following the switch to berotralstat monotherapy, with the greatest improvements in convenience. The transition from injectable prophylactic medication to berotralstat was generally well tolerated. Patients switching to berotralstat monotherapy maintained good control of their HAE symptoms and reported improved treatment satisfaction.
Sections du résumé
BACKGROUND
BACKGROUND
Berotralstat, a first-line, once-daily oral plasma kallikrein inhibitor for long-term prophylaxis of hereditary angioedema (HAE), is an effective and well-tolerated treatment option.
OBJECTIVE
OBJECTIVE
This report summarizes the safety, effectiveness, and impact on treatment satisfaction in patients who switched from injectable long-term prophylactics (LTPs) to oral berotralstat monotherapy (150 mg daily) at US sites in the international open-label APeX-S study.
METHODS
METHODS
APeX-S (ClinicalTrials.gov, NCT03472040) was an open-label Phase 2 study of berotralstat conducted in 22 countries. Here, we focus on APeX-S patients enrolled at US sites who switched from injectable LTPs to berotralstat 150 mg once-daily monotherapy.
RESULTS
RESULTS
Thirty-four patients discontinued lanadelumab (n = 21), subcutaneous C1 esterase inhibitor (n = 11), or intravenous C1 esterase inhibitor (n = 2) and switched to berotralstat 150 mg monotherapy. Vomiting, diarrhea, and upper respiratory tract infection were the most common adverse events (each 11.8%). Mean monthly attack rates were consistently low following the switch to berotralstat. The mean (standard error of the mean) monthly attack rate was 0.29 (0.11) at Month 1, 0.48 (0.15) at Month 6, and 0.58 (0.23) at Month 12. The median attack rate was 0 attacks/month throughout 12 months of treatment. Improvements were observed in the Treatment Satisfaction Questionnaire for Medication from baseline to Month 12 following the switch to berotralstat monotherapy, with the greatest improvements in convenience.
CONCLUSION
CONCLUSIONS
The transition from injectable prophylactic medication to berotralstat was generally well tolerated. Patients switching to berotralstat monotherapy maintained good control of their HAE symptoms and reported improved treatment satisfaction.
Identifiants
pubmed: 38006972
pii: S1081-1206(23)01463-1
doi: 10.1016/j.anai.2023.11.016
pii:
doi:
Banques de données
ClinicalTrials.gov
['NCT03472040']
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
Copyright © 2023. Published by Elsevier Inc.