Hinze Claas H, Speth Fabian, Oommen Prasad T, Haas Johannes-Peter
Department of Pediatric Rheumatology and Immunology, University Hospital Münster, Albert-Schweitzer-Campus 1, Building D3, 48149, Münster, Germany.
Division of Pediatric Rheumatology, University Medicine, Rostock, Germany.
Pediatr Rheumatol Online J. 2018 Jun 20;16(1):38. doi: 10.1186/s12969-018-0256-7.
Juvenile Dermatomyositis (JDM) is a rare pediatric autoimmune disease with broad variations of the individual course. Data on the optimal management are mostly lacking. Currently treatment decisions are often based on experts' opinions. In order to develop consensus-based treatment strategies for JDM in Germany a survey was pursued to analyze the current clinical practice.
An online survey addressing all members of the Society for Pediatric Rheumatology (GKJR) in Germany and Austria and pediatric neurologists with expertise in JDM was performed in February/March of 2016. The questionnaire consisted of 5 case scenarios including diagnostic criteria, treatment of moderate, severe and refractory JDM, using either multiple choice or a 5-point Likert scale. Basic descriptive statistics were used to analyze the findings.
The survey was completed by 60 pediatric rheumatologists and 7 pediatric neurologists experienced in the management of JDM. Typical findings allowing a diagnosis were considered to be: typical skin changes, proximal muscle weakness, MRI findings, elevated muscle enzymes, nailfold capillary changes, presence of calcinosis and muscle biopsy. Regarding induction treatment of moderate/severe JDM: 59%/74% opted for intermittent intravenous methylprednisolone (IVMP) pulse therapy, and 21%/40% for conventional high-dose oral glucocorticoids. Methotrexate (MTX) was the preferred disease-modifying conventional anti-rheumatic drug (cDMARD) for moderate and severe JDM. Regarding the management of refractory moderate or severe JDM, intravenous immune globulins, mycophenolate mofetil and rituximab were preferred treatment options.
There is consensus about the diagnosis of JDM strongly supported by classic clinical and MRI findings. There is great variety in the treatment of JDM in Germany regarding both induction and maintenance therapy. The development of consensus-based treatment strategies for JDM based on harmonization of current clinical practice is essential in order to allow comparative effectiveness research in the future.
幼年皮肌炎（JDM）是一种罕见的儿童自身免疫性疾病，个体病程差异很大。关于最佳治疗方法的数据大多缺乏。目前的治疗决策往往基于专家意见。为了制定德国JDM基于共识的治疗策略，开展了一项调查以分析当前的临床实践。
2016年2月/3月对德国和奥地利儿科风湿病学会（GKJR）的所有成员以及具有JDM专业知识的儿科神经科医生进行了一项在线调查。问卷包括5个病例场景，涵盖诊断标准、中度、重度和难治性JDM的治疗，采用多项选择或5点李克特量表。使用基本描述性统计分析结果。
60名儿科风湿病学家和7名有JDM管理经验的儿科神经科医生完成了调查。认为可作出诊断的典型表现为：典型皮肤改变、近端肌无力、MRI表现、肌肉酶升高、甲襞毛细血管改变、钙质沉着症的存在和肌肉活检。关于中度/重度JDM的诱导治疗：59%/74%选择间歇性静脉注射甲泼尼龙（IVMP）脉冲疗法，21%/40%选择传统高剂量口服糖皮质激素。甲氨蝶呤（MTX）是中度和重度JDM首选的改善病情抗风湿药物（cDMARD）。关于难治性中度或重度JDM的管理，静脉注射免疫球蛋白、霉酚酸酯和利妥昔单抗是首选治疗方案。
JDM的诊断在经典临床和MRI表现的有力支持下已达成共识。德国在JDM的诱导和维持治疗方面存在很大差异。基于当前临床实践的协调制定JDM基于共识的治疗策略对于未来开展比较疗效研究至关重要。
