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ADA2 缺乏症模拟无网状青斑和血管炎的自身免疫性淋巴增生综合征。

Deficiency of ADA2 mimicking autoimmune lymphoproliferative syndrome in the absence of livedo reticularis and vasculitis.

机构信息

Department of Pediatrics, College of Medicine, King Saud University, Riyadh, Saudi Arabia.

Department of Pediatric Hematology/Oncology, King Abdullah Specialist Children's Hospital and King Abdulaziz Medical City, Ministry of National Guard Health Affairs, Riyadh, Saudi Arabia.

出版信息

Pediatr Blood Cancer. 2018 Apr;65(4). doi: 10.1002/pbc.26912. Epub 2017 Dec 22.

Abstract

Adenosine deaminase-2 (ADA2) deficiency (DADA2) is associated with early onset polyarteritis nodosa and vasculopathy. Classic presentation includes livedo reticularis, vasculitis, and stroke. However, the phenotype and disease severity are variable. We present a 5-year-old female who presented with features that mimicked autoimmune lymphoproliferative syndrome (ALPS) in the absence of classic features of DADA2. Exome sequencing identified a novel homozygous splicing variant in ADA2 c.882-2A > G. Patient responded to anti- tumor necrosis factor medication and is in complete remission. Hematologists should be aware of various hematological presentations of DADA2, including ALPS-like disorder, that might lack vasculitis and livedo reticularis to prevent delay in initiating optimal therapy.

摘要

腺苷脱氨酶 2 缺陷症(DADA2)与早发性多发性动脉炎和血管病变相关。其典型表现包括网状青斑、血管炎和中风。然而,其表型和疾病严重程度存在差异。我们介绍了一位 5 岁女性,她在无 DADA2 典型特征的情况下,出现了类似于自身免疫性淋巴增生综合征(ALPS)的特征。外显子组测序发现 ADA2 基因 c.882-2A > G 的新型纯合剪接变异。患者对肿瘤坏死因子药物治疗有反应,完全缓解。血液科医生应注意 DADA2 的各种血液学表现,包括类似于 ALPS 的疾病,其可能缺乏血管炎和网状青斑,以防止延迟开始最佳治疗。

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