腺苷脱氨酶2缺乏:结节性多动脉炎的鉴别诊断具有挑战性。

IF 1.4 Q3 RHEUMATOLOGY
Shakiba Hassanzadeh, Mohammad Bahadoram, Karim Mowla
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引用次数: 1

摘要

腺苷脱氨酶2缺乏症(DADA2)是一种常染色体隐性遗传病,于2014年首次发现。这是一种单基因疾病,由ADA2基因的功能丧失变异引起。腺苷脱氨酶2缺乏累及中小型血管,其临床表现包括结节性多动脉炎(PAN)样特征,如样皮疹、早发性卒中、低γ球蛋白血症、血液学异常和全身性炎症。早期诊断和治疗DADA2至关重要,因为其临床特征可能危及生命,但可能是可治疗的。DADA2的一线治疗选择是肿瘤坏死因子α抑制剂。我们旨在概述DADA2的已知病理生理、临床表现、诊断和治疗。了解DADA2可能有助于更好地诊断、管理和改善DADA2患者的临床结果。然而,需要进一步研究DADA2的基因型-表型关联和确切的病理生理。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Deficiency of adenosine deaminase 2: a challenging differential diagnosis of polyarteritis nodosa.

Deficiency of adenosine deaminase 2 (DADA2) is an autosomal recessive disorder that was first described in 2014. It is a monogenic disease that is caused by loss-of-function variants in the ADA2 gene. Deficiency of adenosine deaminase 2 involves small- and medium-sized vessels and its clinical presentations include polyarteritis nodosa (PAN)-like features such as livedoid rash, early-onset stroke, hypogammaglobulinemia, hematological abnormalities, and systemic inflammation. Early diagnosis and treatment of DADA2 are crucial as the clinical features could be potentially life-threatening but might be treatable. The first-line treatment of choice in DADA2 is tumor necrosis factor α inhibitors. We aimed to provide an overview of the known pathophysiology, clinical presentations, diagnosis, and treatment of DADA2. A clearer knowledge of DADA2 may help to better diagnose, manage, and improve the clinical outcome of DADA2 patients. However, further studies are required to investigate the genotype-phenotype associations and exact pathophysiology of DADA2.

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来源期刊
Reumatologia
Reumatologia Medicine-Rheumatology
CiteScore
2.70
自引率
0.00%
发文量
44
审稿时长
10 weeks
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