Clinical Experience with Berotralstat in Patients with Hereditary Angioedema with Normal C1-Esterase Inhibitor: A Commented Case Series

IF 3.7 3区医学 Q2 ALLERGY

Journal of Asthma and Allergy Pub Date : 2024-02-23 DOI:10.2147/jaa.s445893

Henry J Kanarek, Drew Austin Saville Mutschelknaus

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引用次数: 0

Abstract

Abstract: Hereditary angioedema (HAE) is a rare genetic disorder characterized by potentially life-threatening episodes of swelling. Most HAE cases are caused by deficient (type I) or dysfunctional (type II) C1-esterase inhibitor (C1-INH) protein. However, some patients present with a subtype of HAE that is associated with normal plasma levels of functional C1-INH protein and complement component 4 (HAE-nC1INH). Treatment of HAE-nC1INH is driven by clinical experience as robust clinical trial data to inform treatment decisions are lacking in this population. This retrospective case series assessed clinical features and treatment outcomes in 15 patients with HAE-nC1INH who initiated long-term prophylaxis with oral berotralstat 150 mg once daily as part of their disease management pathway. Most patients were female (93%), with a median age of 49 years. All patients experienced abdominal swelling attacks. On average, patients tried a mean of 4 different treatments for their HAE, including berotralstat. Although most patients associated prophylactic and on-demand medications that target the bradykinin pathway with improvements in the frequency and/or severity of attacks, treatment outcomes varied considerably between patients, highlighting the importance of a personalized approach to disease management. In this case series, berotralstat was an effective prophylactic treatment option in most patients with HAE-nC1INH. Further studies are required to demonstrate the potential efficacy, safety, and impact on quality of life of currently approved HAE therapies in patients with HAE-nC1INH.

Keywords: hereditary angioedema, normal C1-esterase inhibitor, prophylaxis, berotralstat, lanadelumab, plasma-derived C1-esterase inhibitor, abortive therapy

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C1-Esterase抑制剂正常的遗传性血管性水肿患者使用贝罗曲司他的临床经验：评论性病例系列

摘要：遗传性血管性水肿（HAE）是一种罕见的遗传性疾病，其特征是可能危及生命的肿胀发作。大多数 HAE 病例是由 C1-酯酶抑制剂（C1-INH）蛋白缺乏（I 型）或功能障碍（II 型）引起的。然而，有些患者的 HAE 亚型与血浆中正常水平的功能性 C1-INH 蛋白和补体成分 4（HAE-nC1INH）有关。对 HAE-nC1INH 的治疗主要依靠临床经验，因为这类患者缺乏可靠的临床试验数据作为治疗决策的依据。本回顾性系列病例评估了 15 例 HAE-nC1INH 患者的临床特征和治疗效果，这些患者在疾病管理过程中开始长期口服贝罗曲司他 150 毫克，每天一次。大多数患者为女性（93%），中位年龄为 49 岁。所有患者都经历过腹部肿胀发作。患者平均尝试过 4 种不同的 HAE 治疗方法，其中包括贝罗司他。虽然大多数患者在服用针对缓激肽通路的预防性药物和按需药物后，发作频率和/或严重程度有所改善，但不同患者的治疗效果差异很大，这凸显了个性化疾病管理方法的重要性。在本病例系列中，贝罗曲司他是大多数 HAE-nC1INH 患者的有效预防性治疗选择。关键词：遗传性血管性水肿；正常C1-酯酶抑制剂；预防；贝洛曲司他；拉那度单抗；血浆源性C1-酯酶抑制剂；终止治疗

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of Asthma and Allergy Medicine-Immunology and Allergy

CiteScore

5.30

自引率

6.20%

发文量

185

审稿时长

16 weeks

期刊介绍： An international, peer-reviewed journal publishing original research, reports, editorials and commentaries on the following topics: Asthma; Pulmonary physiology; Asthma related clinical health; Clinical immunology and the immunological basis of disease; Pharmacological interventions and new therapies. Although the main focus of the journal will be to publish research and clinical results in humans, preclinical, animal and in vitro studies will be published where they shed light on disease processes and potential new therapies.