Wu Di, Shen Min
Peking Union Medical College Hospital, Beijing, 100730, China.
Clin Rheumatol. 2017 Apr;36(4):965-969. doi: 10.1007/s10067-016-3523-3. Epub 2016 Dec 27.
Muckle-Wells syndrome (MWS) is a rare autoinflammatory disease. This study aimed to report the clinical features and gene variations of the first case series of MWS patients in Chinese population. Four Han Chinese patients were diagnosed with MWS and followed up at our adult clinic for autoinflammatory diseases. All relevant phenotypes and genotypes were collected. All patients were adult male. The median age of disease onset was 4.5 years, and one patient had adult-onset disease. No positive family history was observed. All patients had a remittent disease course. The duration of fever attacks ranged from 0.5 to 7 days. Skin rashes were present in all patients. The other manifestations included polyarthralgia/arthritis (n = 3), oral ulcers (n = 2), conjunctivitis (n = 2), myalgia (n = 2), headache (n = 2), pharyngitis (n = 1), abdominal pain (n = 1), severe sensorineural hearing loss (n = 1), and chronic meningitis with communicating hydrocephalus (n = 1). None of the patients showed evidence of renal amyloidosis. Each patient carried a heterozygous mutation in an NLRP3 gene, including D29V, V70M, T348M, and Q703K, respectively. D29V and V70M variants were novel mutations in exon 1 of NLRP3. All patients had good response to corticosteroids. Our study suggests that MWS could be identified in Chinese population. Our finding of novel mutations in NLRP3 may expand the diversity of MWS.
穆克勒-韦尔斯综合征(MWS)是一种罕见的自身炎症性疾病。本研究旨在报告中国人群中首例MWS患者系列病例的临床特征和基因变异情况。4例汉族患者被诊断为MWS,并在我们的成人自身炎症性疾病门诊接受随访。收集了所有相关的表型和基因型。所有患者均为成年男性。疾病发病的中位年龄为4.5岁,1例患者为成年发病。未观察到阳性家族史。所有患者的病程呈缓解期。发热发作持续时间为0.5至7天。所有患者均出现皮疹。其他表现包括多关节痛/关节炎(n = 3)、口腔溃疡(n = 2)、结膜炎(n = 2)、肌痛(n = 2)、头痛(n = 2)、咽炎(n = 1)、腹痛(n = 1)、重度感音神经性听力损失(n = 1)和伴有交通性脑积水的慢性脑膜炎(n = 1)。所有患者均未显示肾淀粉样变性的证据。每位患者在NLRP3基因中均携带一个杂合突变,分别为D29V、V70M、T348M和Q703K。D29V和V70M变异是NLRP3外显子1中的新突变。所有患者对皮质类固醇均有良好反应。我们的研究表明,中国人群中可识别出MWS。我们在NLRP3中发现的新突变可能会扩大MWS的多样性。