Clinical Features, Treatment, and Outcome of Macrophage Activation Syndrome Complicating Systemic Juvenile Idiopathic Arthritis: A Multinational, Multicenter Study of 362 Patients

作者：Francesca Minoia, Sergio Davì, AnnaCarin Horne, Erkan Demirkaya, Francesca Bovis, Caifeng Li, Kai Lehmberg, Sheila S Weitzman, Antonella Insalaco, Carine Wouters, Susan Shenoi, Graciela Espada, Seza Özen, Jordi Antón, Raju P. Khubchandani, Ricardo A. G. Russo, Priyankar Pal, Özgür Kasapçopur, Päivi Miettunen, Despoina Maritsi, Rosa Merino, Bita Shakoory, Maria Alessio, Vyacheslav Grigoryevich Chasnyk, Helga Sanner, Yijin Gao, Zeng Huasong, Toshiyuki Kitoh, Tadej Avčin, Michel Fischbach, Michael Frosch, Alexei A. Grom, Adam M. Huber, Marija Jelušić, Sujata Sawhney, Yosef Uziel, Nicolino Ruperto, Alberto Martini, Randy Quentin Cron, Angelo Ravelli · 发表于：Arthritis & Rheumatology · 年份：2014 · DOI：10.1002/art.38802 · 被引用次数：435 · 研究领域：Autoimmune and Inflammatory Disorders Research、Adolescent and Pediatric Healthcare、Hemophilia Treatment and Research

OBJECTIVE: To describe the clinical, laboratory, and histopathologic features, current treatment, and outcome of patients with macrophage activation syndrome (MAS) complicating systemic juvenile idiopathic arthritis (JIA). METHODS: In this multinational, multicenter study, pediatric rheumatologists and hemato-oncologists entered patient data collected retrospectively into a web-based database. RESULTS: A total of 362 patients, 22% of whom had MAS at the onset of systemic JIA, were included in the study by 95 investigators from 33 countries. The most frequent clinical manifestations were fever (96%), hepatomegaly (70%), and splenomegaly (58%). Central nervous system dysfunction and hemorrhages were recorded in 35% and 20% of the patients, respectively. Platelet count and liver transaminase, ferritin, lactate dehydrogenase, triglyceride, and d-dimer levels were the sole laboratory biomarkers showing a percentage change of >50% between the pre-MAS visit and MAS onset. Evidence of macrophage hemophagocytosis was found in 60% of the patients who underwent bone marrow aspiration. MAS occurred most frequently in the setting of active underlying disease, in the absence of a specific trigger. Nearly all patients were given corticosteroids, and 61% received cyclosporine. Biologic medications and etoposide were given to 15% and 12% of the patients, respectively. Approximately one-third of the patients required admission to the intensive care unit (ICU), and the mortality rate was 8%. CO...