剖析并发系统性幼年特发性关节炎的巨噬细胞活化综合征的异质性。
Dissecting the heterogeneity of macrophage activation syndrome complicating systemic juvenile idiopathic arthritis.
作者信息
Minoia Francesca, Davì Sergio, Horne AnnaCarin, Bovis Francesca, Demirkaya Erkan, Akikusa Jonathan, Ayaz Nuray A, Al-Mayouf Sulaiman M, Barone Patrizia, Bica Bianca, Bolt Isabel, Breda Luciana, De Cunto Carmen, Enciso Sandra, Gallizzi Romina, Griffin Thomas, Hennon Teresa, Horneff Gerd, Jeng Michael, Kapovic Ageza M, Lipton Jeffrey M, Magni Manzoni Silvia, Rumba-Rozenfelde Ingrida, Magalhaes Claudia Saad, Sewairi Wafaa M, Stine Kimo C, Vougiouka Olga, Weaver Lehn K, Davidsone Zane, De Inocencio Jaime, Ioseliani Maka, Lattanzi Bianca, Tezer Hasan, Buoncompagni Antonella, Picco Paolo, Ruperto Nicolino, Martini Alberto, Cron Randy Q, Ravelli Angelo
机构信息
From the Istituto Giannina Gaslini, Genoa, Italy; Karolinska University Hospital Solna, Stockholm, Sweden; Gulhane Military Medical Faculty, Ankara, Turkey; Royal Children's Hospital, Melbourne, Australia; Bakırkoy Maternity and Children Education and Research Hospital, Istanbul, Turkey; King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia; Policlinico Università di Catania, Catania, Italy; Hospital Universitário Clementino Fraga Filho, Rio de Janeiro, Brazil; Universitaetsklinik fuer Kinderheilkunde, Inselspital, Berne, Switzerland; Ospedale Policlinico, Chieti, Italy; Hospital Italiano de Buenos Aires, Buenos Aires, Argentina; Hospital Infantil de Mexico Federico Gomez, Mexico City, Mexico; University of Messina, Messina, Italy; Carolinas HealthCare System, Charlotte, North Carolina; Women and Children's Hospital, Buffalo, New York, USA; Zentrum fuer Allgemeine Paediatrie und Neonatologie, Sankt Augustin, Germany; Stanford School of Medicine, Palo Alto, California, USA; Department for Immunology and Rheumatology, Children's Hospital of Zagreb, Zagreb, Croatia; Steven and Alexandra Cohen Children's Hospital of New York, New York, New York, USA; IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy; University of Latvia, Riga, Latvia; Hospital das Clínicas, Botucatu, Brazil; King Fahad National Guard Hospital, Riyadh, Saudi Arabia; Arkansas Children's Hospital, Little Rock, Arkansas, USA; Kyriakou Children's Hospital of Athens, Athens, Greece; The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA; Children's University Hospital, Riga, Latvia; Hospital Universitario 12 de Octubre, Madrid, Spain; M. Iashvili Children's Central Clinic, Tbilisi, Georgia; Azienda Ospedaliera Universitaria Ospedali Riuniti, Ancona, Italy; Gazi University, Ankara, Turkey; Università degli Studi di Genova, Genoa, Italy; University of Alabama at Birmingham, Birmingham, Alabama, USA.F. Minoia, MD; S. Davì, MD, Istituto Giannina Gaslini; A. Horne, M
出版信息
J Rheumatol. 2015 Jun;42(6):994-1001. doi: 10.3899/jrheum.141261. Epub 2015 Apr 15.
OBJECTIVE
To seek insights into the heterogeneity of macrophage activation syndrome (MAS) complicating systemic juvenile idiopathic arthritis (sJIA) through the analysis of a large patient sample collected in a multinational survey.
METHODS
International pediatric rheumatologists and hemato-oncologists entered their patient data, collected retrospectively, in a Web-based database. The demographic, clinical, laboratory, histopathologic, therapeutic, and outcome data were analyzed in relation to (1) geographic location of caring hospital, (2) subspecialty of attending physician, (3) demonstration of hemophagocytosis, and (4) severity of clinical course.
RESULTS
A total of 362 patients were included by 95 investigators from 33 countries. Demographic, clinical, laboratory, and histopathologic features were comparable among patients seen in diverse geographic areas or by different pediatric specialists. Patients seen in North America were given biologics more frequently. Patients entered by pediatric hemato-oncologists were treated more commonly with biologics and etoposide, whereas patients seen by pediatric rheumatologists more frequently received cyclosporine. Patients with demonstration of hemophagocytosis had shorter duration of sJIA at MAS onset, higher prevalence of hepatosplenomegaly, lower levels of platelets and fibrinogen, and were more frequently administered cyclosporine, intravenous immunoglobulin (IVIG), and etoposide. Patients with severe course were older, had longer duration of sJIA at MAS onset, had more full-blown clinical picture, and were more commonly given cyclosporine, IVIG, and etoposide.
CONCLUSION
The clinical spectrum of MAS is comparable across patients seen in different geographic settings or by diverse pediatric subspecialists. There was a disparity in the therapeutic choices among physicians that underscores the need to establish uniform therapeutic protocols.
目的
通过对一项多国调查中收集的大量患者样本进行分析，深入了解并发系统性幼年特发性关节炎（sJIA）的巨噬细胞活化综合征（MAS）的异质性。
方法
国际儿科风湿病学家和血液肿瘤学家将他们回顾性收集的患者数据录入一个基于网络的数据库。对人口统计学、临床、实验室、组织病理学、治疗及预后数据进行分析，分析内容涉及：（1）负责治疗的医院地理位置；（2）主治医生的亚专业；（3）噬血细胞现象的表现；（4）临床病程的严重程度。
结果
来自33个国家的95名研究人员共纳入了362例患者。不同地理区域或不同儿科专家诊治的患者在人口统计学、临床、实验室及组织病理学特征方面具有可比性。北美地区的患者更常使用生物制剂。儿科血液肿瘤学家诊治的患者更常接受生物制剂和依托泊苷治疗，而儿科风湿病学家诊治的患者更常接受环孢素治疗。有噬血细胞现象表现的患者在MAS发病时sJIA病程较短，肝脾肿大患病率较高，血小板和纤维蛋白原水平较低，且更常接受环孢素、静脉注射免疫球蛋白（IVIG）和依托泊苷治疗。临床病程严重的患者年龄较大，在MAS发病时sJIA病程较长，临床表现更全面，且更常接受环孢素、IVIG和依托泊苷治疗。
结论
不同地理环境下或不同儿科亚专业医生诊治的患者中，MAS的临床谱具有可比性。医生之间的治疗选择存在差异，这突出了制定统一治疗方案的必要性。
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