目标:我们描述了人口统计学,临床特征,病程,和结节性多动脉炎(PAN)的生存通过国际合作(GLOBAL-PAN)。
方法:从欧洲9个国家的观察队列中招募了1990年至2020年的PAN患者,Japan,和北美。使用欧洲药品管理局分类算法回顾性定义资格。与乙型肝炎病毒相关的PAN患者(n=12)和两种模拟PAN的单基因疾病,缺乏腺苷脱氨酶2酶(n=16)或家族性地中海热(n=11),被排除在外。关于器官受累的数据,复发,疾病相关的损害,和生存进行了分析。
结果:三百五十八例患者(女性:男性比例174:184),包括那些具有系统性PAN(SPAN,n=282)和皮肤PAN(n=76),包括在内。25例是儿科发病。诊断时的平均±SD年龄为44.3±18.1岁。体质症状(71.5%),皮肤受累(70.5%),肌肉骨骼检查结果(69.1%),和神经系统特征(48.0%)是常见表现。在患有sPAN的患者中,胃肠道受累和蛋白尿超过400毫克/天的比例分别为52.2%和11.2%,分别。在中位(四分位距)59.6(99.5)个月的随访期间,48.5%的患者复发.一,sPAN的5年和10年生存率为97.1%,94.0%,和89.0%,分别。sPAN死亡的预测因素包括诊断时年龄≥65岁,诊断时血清肌酐>140μmol/L,胃肠道表现,中枢神经系统(CNS)受累。
结论:PAN的光谱仍然很复杂,多面性疾病。复发是常见的。诊断时年龄≥65岁,血清肌酐>140μmol/L,以及胃肠道和中枢神经系统的参与,是sPAN死亡的独立预测因子。
OBJECTIVE: We describe the demographics, clinical features, disease course, and survival of polyarteritis nodosa (PAN) through an international collaboration (GLOBAL-PAN).
METHODS: Patients with PAN were recruited between 1990 and 2020 from observational cohorts of nine countries across Europe, Japan, and North America. Eligibility was retrospectively defined using the European Medicines Agency classification algorithm. Patients with PAN related to hepatitis B virus (n = 12) and two monogenic diseases mimicking PAN, deficiency of adenosine deaminase 2 enzyme (n = 16) or familial Mediterranean fever (n = 11), were excluded. Data regarding organ involvement, relapse, disease-related damage, and survival were analyzed.
RESULTS: Three hundred fifty-eight patients (female:male ratio 174:184), including those with systemic PAN (sPAN, n = 282) and cutaneous PAN (n = 76), were included. Twenty-five were pediatric onset. Mean ± SD age at diagnosis was 44.3 ± 18.1 years. Constitutional symptoms (71.5%), cutaneous involvement (70.5%), musculoskeletal findings (69.1%), and neurologic features (48.0%) were common manifestations. Among patients with sPAN, gastrointestinal involvement and proteinuria over 400 mg/day were reported in 52.2% and 11.2%, respectively. During a median (interquartile range) 59.6 (99.5) months of follow-up, relapse occurred in 48.5% of patients. One, 5- and 10-year survival rates for sPAN were 97.1%, 94.0%, and 89.0%, respectively. Predictors of death for sPAN included age ≥65 years at diagnosis, serum creatinine at diagnosis >140 μmol/L, gastrointestinal manifestations, and central nervous system (CNS) involvement.
CONCLUSIONS: The spectrum of PAN remains a complex, multifaceted disease. Relapse is common. Age ≥65 years and serum creatinine >140 μmol/L at diagnosis, as well as gastrointestinal and CNS involvement, are independent predictors of death in sPAN.