Department of Immunology, 2nd Faculty of Medicine Charles University and Motol University Hospital, Prague, Czechia,
Department of Immunology, 2nd Faculty of Medicine Charles University and Motol University Hospital, Prague, Czechia.
Int Arch Allergy Immunol. 2021;182(7):642-649. doi: 10.1159/000512933. Epub 2021 Jan 20.
Acquired angioedema with C1 inhibitor deficiency (AAE-C1-INH) is rare but a potentially life-threatening disease. There are no official prevalence data, nor approved therapies for this condition.
In this study, we aimed to collect and analyze clinical data on patients with AAE-C1-INH in the Czech Republic.
We have conducted a retrospective analysis of AAE-C1-INH patients from Czech referral centers for the treatment of hereditary angioedema with C1 inhibitor deficiency. The inclusion criteria involved recurrent episodes of angioedema with the first manifestation at or after the age of 40, negative family history of angioedema, and C1 inhibitor function 50% or less.
A total of 14 patients (7 males and 7 females) met the inclusion criteria for AAE-C1-INH. The median age of the symptom onset was 59.5 years, and the median diagnosis delay was 1 year. The most common clinical manifestation was facial edema (100%) and upper airway swelling (85.7%). All patients responded to the acute attack treatment with icatibant and plasma-derived or recombinant C1 inhibitor concentrate. Lymphoid malignancy was identified in 9 patients (64%), monoclonal gammopathy of uncertain significance in 3 (21%), and in 1 patient autoimmune disease (ulcerative colitis) was considered causative (7%). We were not able to identify any underlying disease only in 1 patient (7%). In 6 of 7 patients (86%) treated for lymphoma, either a reduction in the frequency of angioedema attacks or both angioedema symptoms' disappearance and complement parameter normalization was observed.
The prevalence of AAE-C1-INH in the Czech Republic is about 1:760,000. This rare condition occurs in approximately 8% of the patients with angioedema with C1 inhibitor deficiency. AAE-C1-INH is strongly associated with lymphoproliferative disorders, and treating these conditions may improve the control of angioedema symptoms.
获得性血管性水肿伴 C1 抑制剂缺乏症(AAE-C1-INH)较为罕见,但却是一种潜在的危及生命的疾病。目前尚无该疾病的官方流行率数据或批准的治疗方法。
本研究旨在收集和分析捷克共和国 AAE-C1-INH 患者的临床数据。
我们对捷克遗传性血管性水肿伴 C1 抑制剂缺乏症治疗转诊中心的 AAE-C1-INH 患者进行了回顾性分析。纳入标准为:反复发作的血管性水肿,首次发作年龄在 40 岁或以上,无血管性水肿家族史,C1 抑制剂功能为 50%或更低。
共有 14 名(7 名男性和 7 名女性)患者符合 AAE-C1-INH 的纳入标准。症状发作的中位年龄为 59.5 岁,中位诊断延迟时间为 1 年。最常见的临床表现为面部水肿(100%)和上呼吸道肿胀(85.7%)。所有患者均对急性发作治疗使用艾替班特和血浆衍生或重组 C1 抑制剂浓缩物有反应。9 名患者(64%)诊断为淋巴恶性肿瘤,3 名患者(21%)为意义未明的单克隆丙种球蛋白血症,1 名患者(7%)考虑为自身免疫性疾病(溃疡性结肠炎)。在 1 名患者(7%)中,我们未能确定任何潜在疾病。在 7 名接受淋巴瘤治疗的患者中,有 6 名(86%)患者的血管性水肿发作频率降低,或血管性水肿症状消失和补体参数正常化。
捷克共和国的 AAE-C1-INH 患病率约为 1:760,000。这种罕见疾病在 C1 抑制剂缺乏症所致血管性水肿患者中的发生率约为 8%。AAE-C1-INH 与淋巴增生性疾病密切相关,治疗这些疾病可能改善血管性水肿症状的控制。