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印度南部一家三级护理中心 208 例先天性免疫缺陷患者的特征。

Profile of 208 patients with inborn errors of immunity at a tertiary care center in South India.

机构信息

Pediatric Immunology and Rheumatology, Aster CMI Hospital, Bengaluru, India.

Neuberg Diagnostics, Ahmedabad, India.

出版信息

Clin Exp Med. 2023 Dec;23(8):5399-5412. doi: 10.1007/s10238-023-01225-8. Epub 2023 Oct 28.

Abstract

Primary immune deficiencies or inborn errors of immunity (IEI) are a heterogeneous group of disorders that predispose affected individuals to infections, allergy, autoimmunity, autoinflammation and malignancies. IEIs are increasingly being recognized in the Indian subcontinent. Two hundred and eight patients diagnosed with an IEI during February 2017 to November 2021 at a tertiary care center in South India were included in the study. The clinical features, laboratory findings including microbiologic and genetic data, and treatment and outcome details were analyzed. The diagnosis of IEI was confirmed in a total of 208 patients (198 kindreds) based on relevant immunological tests and/or genetic tests. The male-to-female ratio was 1.8:1. Of the 208 patients, 72 (34.6%) were < 1 yr, 112 (53.8%) were 1-18 years, and 24 (11.5%) were above 18 years. The most common IEI in our cohort was SCID (17.7%) followed by CGD (12.9%) and CVID (9.1%). We also had a significant proportion of patients with DOCK8 deficiency (7.2%), LAD (6.2%) and six patients (2.8%) with autoinflammatory diseases. Autoimmunity was noted in forty-six (22%) patients. Molecular testing was performed in 152 patients by exome sequencing on the NGS platform, and a genetic variant was reported in 132 cases. Twenty-nine children underwent 34 HSCT, and 135 patients remain on supportive therapy such as immunoglobulin replacement and/or antimicrobial prophylaxis. Fifty-nine (28.3%) patients died during the study period, and infections were the predominant cause of mortality. Seven families underwent prenatal testing in the subsequent pregnancy. We describe the profile of 208 patients with IEI, and to the best of our knowledge, this represents the largest data on IEI from the Indian subcontinent reported so far.

摘要

原发性免疫缺陷病或先天性免疫缺陷(IEI)是一组异质性疾病,使受影响的个体易患感染、过敏、自身免疫、自身炎症和恶性肿瘤。IEI 在印度次大陆的发病率越来越高。本研究纳入了 2017 年 2 月至 2021 年 11 月在印度南部一家三级护理中心诊断为 IEI 的 208 例患者。分析了其临床特征、实验室发现(包括微生物学和遗传学数据)以及治疗和预后详情。共有 208 例(198 个家系)患者根据相关免疫学和/或遗传学检查确诊为 IEI。男女比例为 1.8:1。208 例患者中,72 例(34.6%)年龄<1 岁,112 例(53.8%)为 1-18 岁,24 例(11.5%)年龄>18 岁。本队列中最常见的 IEI 是 SCID(17.7%),其次是 CGD(12.9%)和 CVID(9.1%)。我们还发现相当比例的患者存在 DOCK8 缺陷(7.2%)、LAD(6.2%)和 6 例(2.8%)自身炎症性疾病。46 例(22%)患者存在自身免疫。152 例患者通过 NGS 平台进行了外显子组测序,报告了 132 例基因突变。29 例儿童接受了 34 次 HSCT,135 例患者仍接受免疫球蛋白替代和/或抗菌预防等支持性治疗。研究期间,59 例(28.3%)患者死亡,感染是主要死亡原因。随后有 7 个家庭对下一胎进行了产前检测。我们描述了 208 例 IEI 患者的特征,据我们所知,这是迄今为止报告的来自印度次大陆最大的 IEI 数据。

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