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土耳其遗传性血管性水肿初步研究(TURHAPS):首个土耳其遗传性血管性水肿系列研究。

The Turkish Hereditary Angioedema Pilot Study (TURHAPS): the first Turkish series of hereditary angioedema.

机构信息

Department of Medical Genetics, Şişli Etfal Training and Education Hospital, Istanbul, Turkey.

出版信息

Int Arch Allergy Immunol. 2011;156(4):443-50. doi: 10.1159/000323915. Epub 2011 Aug 10.

Abstract

BACKGROUND

No published data presently exist concerning hereditary angioedema (HAE) in Turkey. The aim of the study was to initiate a preliminary multicentric evaluation about HAE and to determine the genetic properties of Turkish patients.

METHODS

Based on records drawn from four medical centers we identified a total of 70 subjects, belonging to 60 unrelated families, fulfilling clinical and laboratory criteria for diagnosis of HAE with C1 inhibitor deficiency. Ten type I patients, and their first-degree relatives, underwent genetic analysis for HAE.

RESULTS

The majority of patients were female (60%), the mean age was 37.7 ± 14.1 years. The mean age at the time of first angioedema symptom was 12.5 ± 9.2 years. Mean time lag between first symptom and diagnosis was 26 ± 14.4 years. All but 3 subjects had HAE type I. Family history of angioedema was present in 75.7% of the cases. Cutaneous swelling was reported by 87.1% of the patients, facial edema by 65%, abdominal symptoms by 74.3% and approximately one half (55.7%) had experienced one or more laryngeal attack. Genetic analysis of 10 families demonstrated that 5 carried a mutation that had never been previously described.

CONCLUSION

We found that the clinical features of Turkish HAE patients were consistent with previously described patterns of this rare disease. The most noteworthy feature identified in the study was a significantly long duration between the first symptom appearance and final diagnosis. Our detection of different mutations in 10 patients confirms the allelic heterogeneity of the disease.

摘要

背景

目前土耳其尚无遗传性血管性水肿(HAE)的相关报道。本研究旨在初步开展多中心评估HAE,并确定土耳其患者的遗传特征。

方法

根据来自 4 个医疗中心的记录,我们共鉴定了 70 名患者,均符合 C1 抑制剂缺乏症所致 HAE 的临床和实验室诊断标准,且属于 60 个无血缘关系的家庭。10 名 I 型患者及其一级亲属接受 HAE 的基因分析。

结果

大多数患者为女性(60%),平均年龄为 37.7±14.1 岁。首次出现血管性水肿症状的平均年龄为 12.5±9.2 岁。首次出现症状至确诊的平均时间间隔为 26±14.4 年。除 3 名患者外,其余均为 I 型 HAE。75.7%的病例有血管性水肿家族史。87.1%的患者有皮肤肿胀,65%有面部水肿,74.3%有腹部症状,约一半(55.7%)有过一次或多次喉头水肿发作。对 10 个家庭的基因分析显示,有 5 个家庭携带以前从未描述过的突变。

结论

我们发现土耳其 HAE 患者的临床特征与该病的已知表现一致。本研究中最显著的特征是首次出现症状至最终确诊的时间明显延长。10 名患者中不同突变的检测证实了该疾病的等位基因异质性。

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