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