Bohm Marek, Gonzalez Fernandez Maria Isabel, Ozen Seza, Pistorio Angela, Dolezalova Pavla, Brogan Paul, Barbano Giancarlo, Sengler Claudia, Klein-Gitelman Marisa, Quartier Pierre, Fasth Anders, Herlin Troels, Terreri Maria Teresa R A, Nielsen Susan, van Rossum Marion A J, Avcin Tadej, Castell Esteban Rodolfo, Foeldvari Ivan, Foell Dirk, Kondi Anuela, Koné-Paut Isabelle, Kuester Rolf-Michael, Michels Hartmut, Wulffraat Nico, Amer Halima Ben, Malattia Clara, Martini Alberto, Ruperto Nicolino
Istituto Giannina Gaslini Pediatria II - Reumatologia, PRINTO, Genova, Italy ; Charles University in Prague, 1st Medical Faculty and General University Hospital in Prague, Prague, Czech Republic.
Istituto Giannina Gaslini Pediatria II - Reumatologia, PRINTO, Genova, Italy.
Pediatr Rheumatol Online J. 2014 May 26;12:18. doi: 10.1186/1546-0096-12-18. eCollection 2014.
Granulomatosis with polyangiitis (GPA), formerly known as Wegener's granulomatosis (WG), belongs to the group of ANCA-associated necrotizing vasculitides. This study describes the clinical picture of the disease in a large cohort of GPA paediatric patients. Children with age at diagnosis ≤ 18 years, fulfilling the EULAR/PRINTO/PRES GPA/WG classification criteria were extracted from the PRINTO vasculitis database. The clinical signs/symptoms and laboratory features were analysed before or at the time of diagnosis and at least 3 months thereafter and compared with other paediatric and adult case series (>50 patients) derived from the literature.
The 56 children with GPA/WG were predominantly females (68%) and Caucasians (82%) with a median age at disease onset of 11.7 years, and a median delay in diagnosis of 4.2 months. The most frequent organ systems involved before/at the time of diagnosis were ears, nose, throat (91%), constitutional (malaise, fever, weight loss) (89%), respiratory (79%), mucosa and skin (64%), musculoskeletal (59%), and eye (35%), 67% were ANCA-PR3 positive, while haematuria/proteinuria was present in > 50% of the children. In adult series, the frequency of female involvement ranged from 29% to 50% with lower frequencies of constitutional (fever, weight loss), ears, nose, throat (oral/nasal ulceration, otitis/aural discharge), respiratory (tracheal/endobronchial stenosis/obstruction), laboratory involvement and higher frequency of conductive hearing loss than in this paediatric series.
Paediatric patients compared to adults with GPA/WG have similar pattern of clinical manifestations but different frequencies of organ involvement.
肉芽肿性多血管炎（GPA），以前称为韦格纳肉芽肿（WG），属于抗中性粒细胞胞浆抗体（ANCA）相关的坏死性血管炎。本研究描述了一大群GPA儿科患者的疾病临床表现。从PRINTO血管炎数据库中提取诊断时年龄≤18岁、符合欧洲抗风湿病联盟（EULAR）/儿科风湿病国际试验组织（PRINTO）/小儿风湿病学会（PRES）GPA/WG分类标准的儿童。在诊断前或诊断时以及此后至少3个月分析临床体征/症状和实验室特征，并与文献中其他儿科和成人病例系列（>50例患者）进行比较。
56例GPA/WG患儿以女性（68%）和白种人（82%）为主，疾病发病的中位年龄为11.7岁，诊断的中位延迟时间为4.2个月。诊断前/诊断时最常受累的器官系统是耳、鼻、喉（91%）、全身症状（不适、发热、体重减轻）（89%）、呼吸系统（79%）、黏膜和皮肤（64%）、肌肉骨骼系统（59%）和眼睛（35%），67%为ANCA-PR3阳性，超过50%的儿童有血尿/蛋白尿。在成人病例系列中，女性受累的频率为29%至50%，全身症状（发热、体重减轻）、耳、鼻、喉（口腔溃疡/鼻溃疡、中耳炎/耳漏）、呼吸系统（气管/支气管内狭窄/阻塞）受累的频率较低，实验室检查受累的频率较低，传导性听力损失的频率高于本儿科系列。
与成人GPA/WG患者相比，儿科患者有相似的临床表现模式，但器官受累的频率不同。
