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伴有波动的神经精神症状、免疫球蛋白 G1 缺乏症和随后发生克罗恩病的患者行全外显子组测序:病例报告。

Whole-exome sequencing in a subject with fluctuating neuropsychiatric symptoms, immunoglobulin G1 deficiency, and subsequent development of Crohn's disease: a case report.

机构信息

Department of Pediatrics, Saint Peter's University Hospital (SPUH), New Brunswick, USA.

Department of Pediatrics, Rutgers University-Robert Wood Johnson Medical School, SPUH, 254 Easton Ave., New Brunswick, NJ, 08901, USA.

出版信息

J Med Case Rep. 2022 May 11;16(1):187. doi: 10.1186/s13256-022-03404-9.

Abstract

BACKGROUND

Mutations or polymorphisms of genes that are associated with inflammasome functions are known to predispose individuals to Crohn's disease and likely affect clinical presentations and responses to therapeutic agents in patients with Crohn's disease. The presence of additional gene mutations/polymorphisms that can modify immune responses may further affect clinical features, making diagnosis and management of Crohn's disease even more challenging. Whole-exome sequencing is expected to be instrumental in understanding atypical presentations of Crohn's disease and the selection of therapeutic measures, especially when multiple gene mutations/polymorphisms affect patients with Crohn's disease. We report the case of a non-Hispanic Caucasian female patient with Crohn's disease who was initially diagnosed with pediatric acute-onset neuropsychiatric syndrome with fluctuating anxiety symptoms at 9 years of age. This patient was initially managed with pulse oral corticosteroid treatment and then intravenous immunoglobulin due to her immunoglobulin G1 deficiency. At 15 years of age, she was diagnosed with Crohn's disease, following onset of acute abdomen. Treatment with oral corticosteroid and then tumor necrosis factor-α blockers (adalimumab and infliximab) led to remission of Crohn's disease. However, she continued to suffer from chronic abdominal pain, persistent headache, general fatigue, and joint ache involving multiple joints. Extensive gastrointestinal workup was unrevealing, but whole-exome sequencing identified two autosomal dominant gene variants: NLRP12 (loss of function) and IRF2BP2 (gain of function). Based on whole-exome sequencing findings, infliximab was discontinued and anakinra, an interleukin-1β blocker, was started, rendering marked improvement of her clinical symptoms. However, Crohn's disease lesions recurred following Yersinia enterocolitis. The patient was successfully treated with a blocker of interleukin-12p40 (ustekinumab), and anakinra was discontinued following remission of her Crohn's disease lesions.

CONCLUSION

Loss-of-function mutation of NRLRP12 gene augments production of interleukin-1β and tumor necrosis factor-α, while gain-of-function mutation of IRF2BP2 impairs cytokine production and B cell differentiation. We propose that the presence of these two autosomal dominant variants caused an atypical clinical presentation of Crohn's disease.

摘要

背景

与炎症小体功能相关的基因的突变或多态性已知会使个体易患克罗恩病,并可能影响克罗恩病患者的临床表现和对治疗药物的反应。存在其他可以改变免疫反应的基因突变/多态性可能会进一步影响临床特征,使克罗恩病的诊断和管理更加具有挑战性。全外显子组测序有望有助于理解克罗恩病的非典型表现和治疗措施的选择,特别是当多个基因突变/多态性影响克罗恩病患者时。我们报告了一例非西班牙裔白种女性克罗恩病患者的病例,该患者最初被诊断为儿科急性发作神经精神综合征,9 岁时出现波动的焦虑症状。该患者最初接受脉冲口服皮质类固醇治疗,然后因免疫球蛋白 G1 缺乏症接受静脉免疫球蛋白治疗。15 岁时,她因急性腹痛发作被诊断为克罗恩病。口服皮质类固醇和肿瘤坏死因子-α 阻滞剂(阿达木单抗和英夫利昔单抗)治疗使克罗恩病缓解。然而,她仍然患有慢性腹痛、持续性头痛、全身疲劳和累及多个关节的关节疼痛。广泛的胃肠道检查结果未发现异常,但全外显子组测序发现了两种常染色体显性基因突变:NLRP12(功能丧失)和 IRF2BP2(功能获得)。基于全外显子组测序结果,停用英夫利昔单抗并开始使用白细胞介素-1β 阻滞剂阿那白滞素,使她的临床症状明显改善。然而,在耶尔森氏菌性肠炎后,克罗恩病病变再次复发。该患者成功接受白细胞介素-12p40 阻滞剂(乌司奴单抗)治疗,在克罗恩病病变缓解后停用阿那白滞素。

结论

NRLRP12 基因的功能丧失性突变增强白细胞介素-1β和肿瘤坏死因子-α的产生,而 IRF2BP2 的功能获得性突变则损害细胞因子的产生和 B 细胞分化。我们提出,这两种常染色体显性变体的存在导致了克罗恩病的非典型临床表现。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/92ca/9092677/ce99c8d82842/13256_2022_3404_Fig1_HTML.jpg

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