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肿瘤坏死因子受体相关自身炎症综合征(TRAPS)的初始表现型:来自欧洲发热/欧洲TRAPS国际注册中心的158例病例系列

The phenotype of TNF receptor-associated autoinflammatory syndrome (TRAPS) at presentation: a series of 158 cases from the Eurofever/EUROTRAPS international registry.

作者信息

Lachmann H J, Papa R, Gerhold K, Obici L, Touitou I, Cantarini L, Frenkel J, Anton J, Kone-Paut I, Cattalini M, Bader-Meunier B, Insalaco A, Hentgen V, Merino R, Modesto C, Toplak N, Berendes R, Ozen S, Cimaz R, Jansson A, Brogan P A, Hawkins P N, Ruperto N, Martini A, Woo P, Gattorno M

机构信息

National Amyloidosis Centre, Royal Free Campus, University College Medical School, London, UK.

Pediatria II, Istituto Giannina Gaslini, Genova, Italy.

出版信息

Ann Rheum Dis. 2014 Dec;73(12):2160-7. doi: 10.1136/annrheumdis-2013-204184. Epub 2013 Aug 21.

Abstract

OBJECTIVE

To evaluate the genetic findings, demographic features and clinical presentation of tumour necrosis factor receptor-associated autoinflammatory syndrome (TRAPS) in patients from the Eurofever/EUROTRAPS international registry.

METHODS

A web-based registry collected retrospective data on patients with TNFRSF1A sequence variants and inflammatory symptoms. Participating hospitals included paediatric rheumatology centres and adult centres with a specific interest in autoinflammatory diseases. Cases were independently validated by experts in the disease.

RESULTS

Complete information on 158 validated patients was available. The most common TNFRSF1A variant was R92Q (34% of cases), followed by T50M (10%). Cysteine residues were disrupted in 27% of cases, accounting for 39% of sequence variants. A family history was present in 19% of patients with R92Q and 64% of those with other variants. The median age at which symptoms began was 4.3 years but 9.1% of patients presented after 30 years of age. Attacks were recurrent in 88% and the commonest features associated with the pathogenic variants were fever (88%), limb pain (85%), abdominal pain (74%), rash (63%) and eye manifestations (45%). Disease associated with R92Q presented slightly later at a median of 5.7 years with significantly less rash or eye signs and more headaches. Children were more likely than adults to present with lymphadenopathy, periorbital oedema and abdominal pains. AA amyloidosis has developed in 16 (10%) patients at a median age of 43 years.

CONCLUSIONS

In this, the largest reported case series to date, the genetic heterogeneity of TRAPS is accompanied by a variable phenotype at presentation. Patients had a median 70 symptomatic days a year, with fever, limb and abdominal pain and rash the commonest symptoms. Overall, there is little evidence of a significant effect of age or genotype on disease features at presentation.

摘要

目的

评估欧洲发热/欧洲肿瘤坏死因子受体相关自身炎症综合征(TRAPS)国际注册研究中患者的基因研究结果、人口统计学特征及临床表现。

方法

基于网络的注册研究收集了有关肿瘤坏死因子受体超家族成员1A(TNFRSF1A)序列变异和炎症症状患者的回顾性数据。参与的医院包括儿科风湿病中心和对自身炎症性疾病有特殊兴趣的成人中心。病例由该疾病领域的专家独立验证。

结果

获得了158例经验证患者的完整信息。最常见的TNFRSF1A变异是R92Q(占病例的34%),其次是T50M(10%)。27%的病例中半胱氨酸残基被破坏,占序列变异的39%。19%携带R92Q变异的患者和64%携带其他变异的患者有家族病史。症状开始出现的中位年龄为4.3岁,但9.1%的患者在30岁以后出现症状。88%的患者发作反复,与致病变异相关的最常见特征为发热(88%)、肢体疼痛(85%)、腹痛(74%)、皮疹(63%)和眼部表现(45%)。与R92Q相关的疾病出现时间稍晚,中位年龄为5.7岁,皮疹或眼部体征明显较少,头痛较多。儿童比成人更易出现淋巴结病、眶周水肿和腹痛。16例(10%)患者发生了AA型淀粉样变性,中位年龄为43岁。

结论

在这个迄今为止报道的最大病例系列中,TRAPS的基因异质性伴随着发病时可变的表型。患者每年中位有70天出现症状,发热、肢体和腹痛以及皮疹是最常见的症状。总体而言,几乎没有证据表明年龄或基因型对发病时的疾病特征有显著影响。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e19b/4251160/f64bab2dcd1c/annrheumdis-2013-204184f01.jpg

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