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婴儿硬膜下血肿、巨颅、皮疹和发育迟缓:NLRP3 中的致病性变异导致 CINCA/NOMID。

Subdural hemorrhage, macrocephaly, rash, and developmental delay in an infant: A pathogenic variant in NLRP3 causes CINCA/NOMID.

机构信息

Safe Child and Adolescent Network, Department of Pediatrics, University of Washington School of Medicine, Seattle Children's Hospital, Seattle, Washington, USA.

Division of Rheumatology, University of Washington School of Medicine, Seattle Children's Hospital, Seattle, Washington, USA.

出版信息

Am J Med Genet A. 2023 Dec;191(12):2825-2830. doi: 10.1002/ajmg.a.63366. Epub 2023 Aug 7.

Abstract

Subdural hemorrhages (SDHs) in children are most often observed in abusive head trauma (AHT), a distinct form of traumatic brain injury, but they may occur in other conditions as well, typically with clear signs and symptoms of an alternative diagnosis. We present a case of an infant whose SDH initially raised the question of AHT, but multidisciplinary evaluation identified multiple abnormalities, including rash, macrocephaly, growth failure, and elevated inflammatory markers, which were all atypical for trauma. These, along with significant cerebral atrophy, ventriculomegaly, and an absence of other injuries, raised concerns for a genetic disorder, prompting genetic consultation. Clinical trio exome sequencing identified a de novo likely pathogenic variant in NLRP3, which is associated with chronic infantile neurological, cutaneous, and articular (CINCA) syndrome, also known as neonatal-onset multisystem inflammatory disease (NOMID). He was successfully treated with interleukin-1 blockade, highlighting the importance of prompt treatment in CINCA/NOMID patients. This case also illustrates how atraumatic cases of SDH can be readily distinguished from AHT with multidisciplinary collaboration and careful consideration of the clinical history and exam findings.

摘要

儿童硬膜下血肿 (SDH) 最常发生于虐待性头部创伤 (AHT),这是一种特殊类型的创伤性脑损伤,但也可能发生在其他情况下,通常有明确的替代诊断的迹象和症状。我们介绍了一例婴儿的病例,其 SDH 最初提出了 AHT 的问题,但多学科评估确定了多种异常,包括皮疹、大头畸形、生长发育不良和炎症标志物升高,这些都与创伤无关。这些情况,加上明显的脑萎缩、脑室扩大和其他损伤的缺失,引起了对遗传疾病的关注,促使进行了遗传咨询。临床三联体外显子组测序发现 NLRP3 中一个新生的可能致病性变异,该变异与慢性婴儿神经皮肤关节(CINCA)综合征(也称为新生儿发病多系统炎症性疾病[NOMID])有关。他成功地接受了白细胞介素-1 阻断治疗,突出了在 CINCA/NOMID 患者中及时治疗的重要性。这个病例还说明了在多学科合作和仔细考虑临床病史和检查结果的情况下,如何容易地区分无创伤性的 SDH 和 AHT。

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