Innovative Research Design to Meet the Challenges of Clinical Trials for Juvenile Dermatomyositis

被引：6

作者：

Rosina, Silvia ^{[1
]}

Varnier, Giulia Camilla ^{[1
]}

Mazzoni, Marta ^{[1
]}

Lanni, Stefano ^{[2
]}

Malattia, Clara ^{[1
,2
]}

Ravelli, Angelo ^{[1
,2
]}

机构：

[1] Univ Genoa, Genoa, Italy

[2] Ist Giannina Gaslini, Clin Pediat & Reumatol, Via G Gaslini 5, I-16147 Genoa, Italy

来源：

CURRENT RHEUMATOLOGY REPORTS | 2018年 / 20卷 / 05期

关键词：

Juvenile dermatomyositis; Idiopathic inflammatory myopathies; Outcome measures; Randomized clinical trials; Disease remission; IDIOPATHIC INFLAMMATORY MYOPATHIES; DISEASE-ACTIVITY SCORE; MYOSITIS-SPECIFIC AUTOANTIBODIES; ALLIANCE CONSENSUS CONFERENCE; CUTANEOUS ASSESSMENT-TOOL; INTERNATIONAL CONSENSUS; DEFINING CRITERIA; ADULT POLYMYOSITIS; PROGNOSTIC-FACTORS; INACTIVE DISEASE;

D O I：

10.1007/s11926-018-0734-4

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Purpose of Review This paper aims to provide a summary of the recent therapeutic advances and the latest research on outcome measures for clinical trials in juvenile dermatomyositis (JDM). Recent Findings Recent randomized controlled trials (RCTs) have demonstrated the superiority of the combination of prednisone with methotrexate over other conventional therapies and the potential effectiveness of rituximab in refractory cases. A multinational project has led to develop new criteria for the definition of minimal, moderate, and major improvement in future JDM clinical trials. This effort has been paralleled by the establishment of criteria for clinically inactive disease. The validation of the first composite disease activity score for JDM is in progress. Summary The new outcome measures will increase the reliability of assessment of clinical response in JDM clinical trials and foster future multinational RCTs aimed to investigate novel treatment strategies for refractory forms of JDM.

引用

页数：8

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