Suppr超能文献

由凝血因子XII基因错义突变引起的遗传性血管性水肿:临床特征、触发因素及治疗

Hereditary angioedema caused by missense mutations in the factor XII gene: clinical features, trigger factors, and therapy.

作者信息

Bork Konrad, Wulff Karin, Hardt Jochen, Witzke Günther, Staubach Petra

机构信息

Department of Dermatology, Johannes Gutenberg University, Langenbeckstr 1, Mainz 55131, Germany.

出版信息

J Allergy Clin Immunol. 2009 Jul;124(1):129-34. doi: 10.1016/j.jaci.2009.03.038. Epub 2009 May 27.

Abstract

BACKGROUND

Hereditary angioedema caused by mutations in the factor XII gene is a recently described disease entity that occurs mainly in women. It differs from hereditary angioedema caused by C1 inhibitor deficiency.

OBJECTIVE

To assess the clinical symptoms, factors triggering acute attacks, and treatments of this disease.

METHODS

Thirty-five female patients with hereditary angioedema and the factor XII mutations p.Thr309Lys and p.Thr309Arg who came from 13 unrelated families were studied. The observation period was 8.4 years on average (range, 2-26 years).

RESULTS

Patients had on average 12.7 +/- 7.9 angioedema attacks per year. Recurrent facial swellings occurred in all patients; skin swellings other than facial, abdominal pain attacks, tongue swellings, and laryngeal edema occurred less frequently. Some factors that triggered angioedema attacks were trauma, physical pressure, and emotional stress. Clinical symptoms started mainly after intake of oral contraceptives (17 women) or pregnancy (3 women). Exacerbation of the symptoms occurred after oral contraceptive use (8 women), pregnancy (7 women), hormone replacement therapy (3 women), intake of angiotensin-converting enzyme inhibitors (2 women), and an angiotensin 1 receptor blocker (1 woman). Effective treatments included C1 inhibitor concentrate for angioedema attacks (6 women) and, for prophylaxis, progesterone (8 women), danazol (2 women), and tranexamic acid (1 woman). No difference between mutation p.Thr309Arg and p.Thr309Lys was found.

CONCLUSIONS

Facial swelling is a cardinal symptom of this condition. Estrogens may have a great influence, but this influence is highly variable. Various treatment options are available.

摘要

背景

由凝血因子 XII 基因突变引起的遗传性血管性水肿是一种最近描述的疾病实体,主要发生于女性。它与由 C1 抑制剂缺乏引起的遗传性血管性水肿不同。

目的

评估该疾病的临床症状、触发急性发作的因素及治疗方法。

方法

对来自 13 个无关家庭的 35 名患有遗传性血管性水肿且存在凝血因子 XII 基因 p.Thr309Lys 和 p.Thr309Arg 突变的女性患者进行研究。观察期平均为 8.4 年(范围为 2 - 26 年)。

结果

患者每年平均发生 12.7±7.9 次血管性水肿发作。所有患者均出现复发性面部肿胀;面部以外的皮肤肿胀、腹痛发作、舌头肿胀和喉头水肿发生频率较低。一些触发血管性水肿发作的因素包括外伤、物理压力和情绪应激。临床症状主要在服用口服避孕药后(17 名女性)或怀孕后(3 名女性)开始出现。在使用口服避孕药后(8 名女性)、怀孕后(7 名女性)、激素替代疗法后(3 名女性)、摄入血管紧张素转换酶抑制剂后(2 名女性)以及使用血管紧张素 1 受体阻滞剂后(1 名女性)症状加重。有效的治疗方法包括用于血管性水肿发作的 C1 抑制剂浓缩物(6 名女性),以及用于预防的孕激素(8 名女性)、达那唑(2 名女性)和氨甲环酸(1 名女性)。未发现 p.Thr309Arg 和 p.Thr309Lys 突变之间存在差异。

结论

面部肿胀是这种疾病的主要症状。雌激素可能有很大影响,但这种影响差异很大。有多种治疗选择。

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验