长期接受预防性治疗的患者中的遗传性血管性水肿发作:系统回顾。

IF 8.4 2区 医学 Q1 ALLERGY
Clinical Reviews in Allergy & Immunology Pub Date : 2024-12-01 Epub Date: 2024-11-07 DOI:10.1007/s12016-024-09006-1
Hilary J Longhurst, Mauro Cancian, Vesna Grivcheva-Panovska, Majed Koleilat, Markus Magerl, Sinisa Savic, Marcin Stobiecki, Raffi Tachdjian, Bridget Healy, Christopher M Yea, Paul K Audhya, Laurence Bouillet
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引用次数: 0

摘要

长期预防疗法(LTP)已被证明可以降低遗传性血管性水肿(HAE)的发作频率;然而,接受 LTP 治疗的患者的发作特征尚未得到很好的描述。本系统性综述旨在评估在接受 LTP 时发作的 I/II 型 HAE(HAE-C1INH)患者的比例、这些发作的特征以及相关的按需治疗使用情况。我们在 PubMed 上进行了系统性检索,以确定报告 HAE-C1INH 患者使用血浆源性 C1 抑制剂 (pdC1INH)、拉那度单抗、贝洛曲司他、雄激素或抗纤维蛋白溶解剂进行 LTP 治疗的研究。45项主要研究符合纳入标准。在3期试验中,皮下注射pdC1INH 60 IU/kg,每周两次,16周时无发作率为40%;拉那度单抗300 mg,每两周一次,6个月时无发作率为44%(稳态[第70-182天]期间为77%);贝罗曲司他150 mg与安慰剂相比,24周时无发作率无差异。据 3 期研究报告,皮下注射和静脉注射 pdC1INH 与安慰剂相比,平均发作严重程度较低。对于拉那珠单抗和贝罗曲司他,外周发作的预防性治疗效果比腹部和喉部发作更明显。在观察性和干预性研究中,喉部发作占所有发作的2%-7%,与接受的LTP药物无关。在出现 LTP 的情况下,49%-94% 的发作采用了按需治疗。总之,接受 LTP 治疗的患者会在包括喉部在内的所有解剖部位发作。大多数发作都接受了按需治疗,但结果尚未报告。对于所有HAE-C1INH患者来说,按需治疗仍然至关重要。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Hereditary Angioedema Attacks in Patients Receiving Long-Term Prophylaxis: A Systematic Review.

Long-term prophylaxis (LTP) has been shown to reduce the frequency of hereditary angioedema (HAE) attacks; however, attacks occurring in patients receiving LTP have not been well characterized. The objective of this systematic review was to evaluate the proportion of type I/II HAE (HAE-C1INH) patients who experience attacks while receiving LTP, the characteristics of these attacks, and associated on-demand therapy use. A systematic search was conducted in PubMed to identify studies reporting LTP use with plasma-derived C1 inhibitor (pdC1INH), lanadelumab, berotralstat, androgens, or antifibrinolytics in patients with HAE-C1INH. Forty-five primary studies met the inclusion criteria. In phase 3 trials, attack-free rates were 40% for subcutaneous pdC1INH 60 IU/kg twice weekly at 16 weeks, and 44% for lanadelumab 300 mg every second week at 6 months (77% during steady-state [days 70-182]); there was no difference in attack-free rate for berotralstat 150 mg versus placebo at 24 weeks. Phase 3 studies reported a lower average attack severity with subcutaneous and intravenous pdC1INH versus placebo. With lanadelumab and berotralstat, the prophylactic treatment effect was more pronounced in peripheral attacks than in abdominal and laryngeal attacks. Laryngeal attacks accounted for 2%-7% of all attacks in observational and interventional studies, regardless of the LTP agent received. On-demand therapy was used in 49%-94% of attacks occurring in the presence of LTP. In conclusion, patients receiving LTP experienced attacks in all anatomic locations, including the larynx. Most attacks were treated with on-demand therapy, although outcomes were not reported. Access to on-demand therapy remains essential for all people with HAE-C1INH.

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来源期刊
CiteScore
22.30
自引率
1.10%
发文量
58
审稿时长
6-12 weeks
期刊介绍: Clinical Reviews in Allergy & Immunology is a scholarly journal that focuses on the advancement of clinical management in allergic and immunologic diseases. The journal publishes both scholarly reviews and experimental papers that address the current state of managing these diseases, placing new data into perspective. Each issue of the journal is dedicated to a specific theme of critical importance to allergists and immunologists, aiming to provide a comprehensive understanding of the subject matter for a wide readership. The journal is particularly helpful in explaining how novel data impacts clinical management, along with advancements such as standardized protocols for allergy skin testing and challenge procedures, as well as improved understanding of cell biology. Ultimately, the journal aims to contribute to the improvement of care and management for patients with immune-mediated diseases.
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