血管炎与腺苷脱氨酶2缺乏相关:在behet病和自身炎症之间的十字路口。一个观点。

IF 16.4 1区 化学 Q1 CHEMISTRY, MULTIDISCIPLINARY
A Colangelo, F Tromby, G Cafaro, R Gerli, E Bartoloni, C Perricone
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引用次数: 0

摘要

腺苷脱氨酶2缺乏症(DADA2)是一种罕见的单基因血管病变,由ADA2(前身为CECR1(猫眼综合征染色体1区)基因的纯合或复合杂合突变导致功能丧失。DADA2表型具有广泛的异质性,患者可能表现为发热、体重减轻、网状/总状斑、手指缺血、皮肤溃疡、周围神经病变、腹痛、肠穿孔、门脉或肾源性高血压。更具体的表现包括早发性缺血性或出血性中风,轻度免疫缺陷和低γ -血红蛋白血症,细胞减少症和视力障碍。在此,我们提出一个年轻男性与DADA2相关的血管炎的病例。HLA-B51的存在和该患者的临床特征提出了ADA2缺乏症、behet病和nod2相关疾病之间的相似性问题。这种罕见的单基因疾病的治疗是具有挑战性的,并且基于小病例系列。该患者的长期经验证明了强的松逐渐减少的困难和缺乏令人满意的治疗策略。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Vasculitis associated with adenosine deaminase 2 deficiency: at the crossroads between Behçet's disease and autoinflammation. A viewpoint.

Adenosine deaminase 2 deficiency (DADA2) is a rare monogenic vasculopathy caused by loss-of-function homozygous or compound heterozygous mutations in ADA2, formerly CECR1 (cat eye syndrome chromosome region 1) gene. The DADA2 phenotype is widely heterogeneous, and patients may present with fever, weight loss, livedo reticularis/racemosa, digital ischemia, cutaneous ulceration, peripheral neuropathy, abdominal pain, bowel perforation, and portal or nephrogenic hypertension. More specific manifestations include early-onset ischemic or hemorrhagic stroke, mild immunodeficiency and hypogammaglobinemia, cytopenia, and vision disturbances. Herein, we present the case of a young male with vasculitis associated with DADA2. The presence of HLA-B51 and the clinical features of this patient raised the question of similarities between ADA2 deficiency, Behçet's disease, and NOD2-associated diseases. Treatment of this rare monogenic disease is challenging and based on small case series. The long-term experience of this patient proved the difficulties of prednisone tapering and the lack of satisfactory therapeutic strategies.

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来源期刊
Accounts of Chemical Research
Accounts of Chemical Research 化学-化学综合
CiteScore
31.40
自引率
1.10%
发文量
312
审稿时长
2 months
期刊介绍: Accounts of Chemical Research presents short, concise and critical articles offering easy-to-read overviews of basic research and applications in all areas of chemistry and biochemistry. These short reviews focus on research from the author’s own laboratory and are designed to teach the reader about a research project. In addition, Accounts of Chemical Research publishes commentaries that give an informed opinion on a current research problem. Special Issues online are devoted to a single topic of unusual activity and significance. Accounts of Chemical Research replaces the traditional article abstract with an article "Conspectus." These entries synopsize the research affording the reader a closer look at the content and significance of an article. Through this provision of a more detailed description of the article contents, the Conspectus enhances the article's discoverability by search engines and the exposure for the research.
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