Silvia Rosina1, Giulia Camilla Varnier1, Marta Mazzoni1, Stefano Lanni2, Clara Malattia1,2, Angelo Ravelli3,4. 1. Università degli Studi di Genova, Genoa, Italy. 2. Clinica Pediatrica e Reumatologia, Istituto Giannina Gaslini, Via G. Gaslini 5, 16147, Genoa, Italy. 3. Università degli Studi di Genova, Genoa, Italy. angeloravelli@gaslini.org. 4. Clinica Pediatrica e Reumatologia, Istituto Giannina Gaslini, Via G. Gaslini 5, 16147, Genoa, Italy. angeloravelli@gaslini.org.
Abstract
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. 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.
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. 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.
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