Division of Rheumatology, Allergy and Immunology, Department of Medicine, University of California San Diego, La Jolla, California.
Asthma & Allergy Associates P.C., Colorado Springs, Colorado.
Ann Allergy Asthma Immunol. 2024 Apr;132(4):505-511.e1. doi: 10.1016/j.anai.2023.11.016. Epub 2023 Nov 24.
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.
To summarize the safety, effectiveness, and impact on treatment satisfaction in patients who switched from injectable long-term prophylactics to oral berotralstat monotherapy (150 mg daily) at US sites in the international open-label APeX-S study.
APeX-S was an open-label, Phase II study of berotralstat conducted in 22 countries. Here, we focus on APeX-S patients enrolled at US sites who switched from injectable long-term prophylactics to berotralstat 150 mg once-daily monotherapy.
A total of 34 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 after the switch to berotralstat. The mean (SEM) 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 attack/mo throughout 12 months of treatment. Improvements were observed in the Treatment Satisfaction Questionnaire for Medication from baseline to Month 12 after 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.
ClinicalTrials.gov Identifier: NCT03472040.
Berotralstat 是一种一线、每日一次、口服血浆激肽释放酶抑制剂，用于遗传性血管水肿（HAE）的长期预防，是一种有效且耐受良好的治疗选择。
总结美国研究地点参与国际开放标签 APeX-S 研究的患者从注射用长期预防药物转为口服贝罗曲肽单药治疗（每日 150mg）的安全性、有效性和对治疗满意度的影响。
APeX-S 是一项关于贝罗曲肽的开放标签、II 期研究，在 22 个国家进行。在这里，我们重点关注在美国研究地点入组的患者，他们从注射用长期预防药物转为贝罗曲肽 150mg 每日一次单药治疗。
共有 34 名患者停用拉那芦单抗（n=21）、皮下 C1 酯酶抑制剂（n=11）或静脉内 C1 酯酶抑制剂（n=2）并转为贝罗曲肽 150mg 单药治疗。最常见的不良事件为呕吐、腹泻和上呼吸道感染（各 11.8%）。转为贝罗曲肽治疗后，每月发作率持续较低。转为贝罗曲肽治疗后第 1、6 和 12 个月的平均每月发作率分别为 0.29（0.11）、0.48（0.15）和 0.58（0.23）。12 个月治疗期间，中位发作率为 0 次/月。转为贝罗曲肽单药治疗后，从基线到第 12 个月，治疗药物满意度问卷评分均有改善，便利性的改善最大。
从注射预防药物转为贝罗曲肽通常可耐受良好。转为贝罗曲肽单药治疗的患者保持了对 HAE 症状的良好控制，并报告治疗满意度提高。
ClinicalTrials.gov 标识符：NCT03472040。
