Nakano Naoko, Mori Masaaki, Umebayashi Hiroaki, Iwata Naomi, Kobayashi Norimoto, Masunaga Kenji, Imagawa Tomoyuki, Murata Takuji, Kinjo Noriko, Nagai Kazushige, Miyoshi Mari, Takei Syuji, Yokota Shumpei, Ishii Eiichi
a Department of Pediatrics , Ehime University Graduate School of Medicine , Ehime , Japan.
b Department of Lifetime Clinical Immunology, Graduate School of Medical and Dental Sciences , Tokyo Medical and Dental University , Tokyo , Japan.
Mod Rheumatol. 2018 Jul;28(4):697-702. doi: 10.1080/14397595.2017.1404700. Epub 2017 Dec 8.
Primary systemic vasculitis (PSV) is a rare disorder in children and difficult to distinguish from other diseases. However, appropriate diagnosis and prompt treatment will affect on the morbidity and mortality of intractable PSV. In this study, we conducted a nationwide survey in Japan, to clarify epidemiology and clinical outcome of PSV.
We had sent survey questionnaires to most of the Japanese institutions that employed pediatricians, requesting the number of patients with refractory PSV who were diagnosed and treated between 2007 and 2011. Respondents were asked to provide detailed information on the clinical and laboratory features of each case they had managed. Those with Kawasaki disease or Henoch-Shönlein purpura vasculitis (IgA vasculitis) were excluded.
Of all the institutions surveyed, 1123 (37.3%) patients responded, finally, total of 49 patients with intractable PSV, defined by those with resistant to treatment and steroid-dependent, or with any complication associated with prognosis, were selected. The diagnosis was Takayasu arteritis in 31, polyarteritis nodosa in 11, granulomatosis with polyangitis in 2, microscopic polyangitis in 1, and ANCA negative microscopic polyangitis in 1. In those with Takayasu arteritis, 67% were treated with an immunosuppressive agent, 22% with biological modifiers, and 16% with surgical procedures. In other types of disease, 88% of the patients were treated with an immunosuppressive agent, and 12% with biological modifiers. Two with Takayasu arteritis died being terminally ill.
This nationwide survey establishes the heterogeneous characteristics of PSV in children. Although questionnaire-based, the results of our analysis should be useful in planning prospective studies to identify the most effective therapy for each subtype of multifaceted disease.
原发性系统性血管炎（PSV）在儿童中是一种罕见疾病，且难以与其他疾病区分开来。然而，恰当的诊断和及时的治疗会影响难治性PSV的发病率和死亡率。在本研究中，我们在日本开展了一项全国性调查，以阐明PSV的流行病学和临床结局。
我们向日本大多数雇佣儿科医生的机构发送了调查问卷，询问2007年至2011年期间诊断和治疗的难治性PSV患者数量。要求受访者提供他们所管理的每个病例的临床和实验室特征的详细信息。患有川崎病或过敏性紫癜性血管炎（IgA血管炎）的患者被排除在外。
在所有接受调查的机构中，1123名（37.3%）患者做出了回应，最终，共选出49例难治性PSV患者，这些患者被定义为对治疗耐药、依赖类固醇或伴有任何与预后相关的并发症。诊断为大动脉炎的有31例，结节性多动脉炎的有11例，肉芽肿性多血管炎的有2例，显微镜下多血管炎的有1例，ANCA阴性显微镜下多血管炎的有1例。在大动脉炎患者中，67%接受了免疫抑制剂治疗，22%接受了生物调节剂治疗，16%接受了外科手术治疗。在其他类型的疾病中，88%的患者接受了免疫抑制剂治疗，12%接受了生物调节剂治疗。两名大动脉炎患者在临终时死亡。
这项全国性调查确定了儿童PSV的异质性特征。尽管基于问卷调查，但我们的分析结果对于规划前瞻性研究以确定针对这种多方面疾病各亚型最有效的治疗方法应该是有用的。
