Recombinant human C1 esterase inhibitor for acute hereditary angioedema attacks with upper airway involvement.
Recombinant human C1 esterase inhibitor for acute hereditary angioedema attacks with upper airway involvement.
复制标题
重组人 C1 酯酶抑制剂，用于治疗累及上呼吸道的急性遗传性血管性水肿发作。
DOI：
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复制
发表时间：
2017
影响因子：
2.8
通讯作者：
A. Relan
中科院分区：
文献类型：
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作者：
M. Riedl;H. H. Li;M. Cicardi;J. Harper;A. Relan
背景
重组人C1酯酶抑制剂（rhC 1-INH）被批准用于治疗青少年和成人的遗传性血管性水肿（HAE）。涉及上呼吸道的HAE发作可能危及生命，目前关于这些类型发作的rhC 1-INH给药数据有限。
目的
目的评价重组人C1-INH治疗急性HAE并上呼吸道受累的有效性和安全性。
方法
来自三项开放标签扩展临床试验的数据的汇总分析检查了rhC 1-INH治疗急性HAE发作伴上呼吸道受累。根据呼吸或吞咽症状的严重程度，回顾性地确定了患有急性上呼吸道HAE发作并接受rhC 1-INH的功能性血浆C1酯酶抑制剂<正常值50%的患者。主要终点为至开始缓解的时间（连续两个时间点总体视觉模拟量表评分[0-100 mm]较基线降低≥20 mm的时间[持续性]）。
结果
在用rhC 1-INH治疗的683例急性HAE发作中，包括45例上呼吸道受累发作的数据。至症状开始缓解的中位时间为67分钟（95%置信区间，60-120分钟），发作次数或基线呼吸或吞咽症状严重程度无差异。大多数发作（91.1%）在rhC 1-INH治疗后4小时内开始缓解。所有发作均在不需要任何额外药物的情况下消退，并且没有患者需要插管或气管切开术。rhC 1-INH治疗耐受性良好，未在1例以上患者中报告不良事件（HAE报告为不良事件除外[n = 2]）。
结论
临床试验数据的汇总分析支持rhC 1-INH治疗急性HAE发作伴上呼吸道受累的疗效。
BACKGROUND
Recombinant human C1 esterase inhibitor (rhC1-INH) is approved for treatment of hereditary angioedema (HAE) in adolescents and adults. HAE attacks that involve the upper airway can be life threatening, and data on the administration of rhC1-INH for these types of attacks are currently limited.
OBJECTIVE
To evaluate the efficacy and safety of rhC1-INH for treatment of acute HAE attacks with upper airway involvement.
METHODS
A pooled analysis of data from three clinical trials with open-label extensions examined rhC1-INH for treatment of acute HAE attacks with upper airway involvement. Patients with functional plasma C1 esterase inhibitor <50% of normal who had experienced an acute upper airway HAE attack and received rhC1-INH were identified retrospectively based on severity of breathing or swallowing symptoms. The primary end point was the time to beginning of relief (time at which the overall visual analog scale score [0-100 mm] decreased from baseline by ≥20 mm for two consecutive time points [persistence]).
RESULTS
Of 683 acute HAE attacks treated with rhC1-INH, data for 45 attacks with upper airway involvement were included. The median time to the beginning of symptom relief was 67 minutes (95% confidence interval, 60-120 minutes) and did not differ by attack number or by baseline breathing or swallowing symptom severity. Most attacks (91.1%) achieved the beginning of relief within 4 hours of rhC1-INH treatment. All attacks resolved without the need for any additional medication, and no patients required intubation or tracheostomy. Treatment with rhC1-INH was well tolerated, with no adverse events reported in more than one patient (except HAE reported as an adverse event [n = 2]).
CONCLUSION
This pooled analysis of clinical trial data supports the efficacy of rhC1-INH for treatment of acute HAE attacks with upper airway involvement.